Podcasts about kyowa kirin

'One FM' by One MSL strives to connect voices within the global Field Medical community.For episode 8 Helen was joined by Andrew Hewitson, Head of Training & Capability Building - Global Medical Affairs Excellence at Kyowa Kirin.If you would like to feature on a future episode, please email community@onemsl.comhttps://www.onemsl.com/NB: The views expressed in this podcast episode are those of the individuals participating & do not represent Kyowa Kirin.

This program is supported by educational grants from Amicus Therapeutics, Inc. and Chiesi USA Inc.Fabry disease is an inherited lysosomal storage disease caused by mutations in the GLA gene, disrupting the function of the enzyme, α-galactosidase. This results in the accumulation of globotriaosylceramide (GL-3) and its deacylated form, globotriaosylsphingosine (lyso-GL-3), leading to progressive disruption of multiple organ systems. There are currently three treatment options available for Fabry disease, including two enzyme replacement therapies, agalsidase beta and pegunigalsidase alfa, and a chaperone therapy, migalastat. There are also other treatments in development (e.g., gene therapy, other enzyme replacement therapies) and some that are available in other countries (e.g., agalsidase alfa). Due to the small patient population and variability in Fabry disease severity, it is challenging to develop properly powered, placebo-controlled clinical trials. As such, data shared at conferences like WORLDSymposium 2025 are crucial for guiding best practices in this disease area. This program, led by Dr. Eric Wallace, provides a summary of clinically relevant data presented at WORLDSymposium 2025 that can enhance the care of patients with Fabry disease.  Target AudienceThis activity has been designed to meet the educational needs of physicians specializing in neurology, nephrology, cardiology, gastroenterology, ophthalmology, and dermatology. Other members of the care team may also participate.Learning ObjectivesAfter participating in the activity, learners should be better able to: Describe the latest research being presented to better manage individuals with Fabry disease and its clinical relevance.Eric Wallace, MDProfessor of MedicineDepartment of NephrologyUniversity of Alabama Medical SchoolDisclosure StatementAccording to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated.Disclosure of relevant financial relationships are as follows:Faculty Educator/PlannerDr. Wallace discloses the following relevant financial relationships with ineligible companies:Advisory Board Consultant: Sanofi-Genzyme, Chiesi, Kyowa Kirin, Sangamo, NateraGrant/Research Support: Sanofi-Genzyme, Chiesi, Uniqure, Idorsia, Amicus Other Planners for this activity have no relevant financial relationships with any ineligible companies.This activity will review off-label or investigational information.The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information.Accreditation and Credit DesignationIn support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is Jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.PhysiciansAmerican Academy of CME, Inc., designates this enduring material for a maximum of 0.50 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Other HCPsOther members of the care team will receive a certificate of participation.There are no fees to participate in the activity.  Participants must review the activity information including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the pre and post-program assessments. Your certificate will be emailed to you within 30 days.Hardware/Software RequirementsWindows Requirements: • Operating system: Windows XP Service Pack 2 or later • Browser: Internet Explorer 7 or later, Mozilla Firefox 2.5 or later • Internet connection: DSL, cable modem, or other high-speed connectionMacintosh Requirements: • Operating system: Mac OS X v10.3 or later • Browser: Mozilla Firefox 2.5 or later • Internet connection: DSL, cable modem, or other high-speed connectionPrivacyFor more information about the American Academy of CME privacy policy, please access http://www.academycme.org/privacy.htm  For more information about CheckRare's privacy policy, please access https://checkrare.com/privacy/ContactFor any questions, please contact: CEServices@academycme.orgCopyright© 2025. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare CE grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).

German is a practitioner of "mixed cognitive arts" building modern brands across a full spectrum of categories (with a leaning toward health, pharma, wellness, beauty, finance, insurance, food, hospitality, sports and automotive).A Silicon Valley-trained entrepreneur and thought leader applying ingenuity and leading methodologies to simplify marketing challenges using human insights, cultural trends, lateral thinking and new technologies.A passionate advocate for anthropology bringing ethnographies, artificial intelligence, expert crowdsourcing and cultural computing to advertising, media, innovation and corporate strategy. A client-savvy executive building long-lasting client relationships and driving high-profile pitch wins and account retention for agencies.A visionary team architect mentoring talent, rejuvenating culture and shaping the future of the Strategy & Planning discipline.Feeling at home in both disruptive and mainstream agencies with a preference for the former.Brands: Procter & Gamble, Unilever, Intel, NASDAQ, Ask.com, Creditcards.com, Ely Lilly, AMGEN, Novartis, Pfizer, Otsuka, Kyowa Kirin, Ipsen, Stryker, Foundation Medicine, National Jewish Health, Weight Watchers, Bank of America/Merrill Lynch, American Family Insurance, Emblem Insurance, Volvo, Volkswagen, Honda, Lufthansa, Expedia, L'Oreal, Estee Lauder, Erno Laszlo, Victoria's Secret, RadioShack, Best Buy, CVS, Burger King, Domino's Pizza, Wendy's, Miller Coors, Brown Forman, Kahlua, Nike, Pearl Izumi.

*This month in partnership with ⁠Choroideremia Research Foundation⁠*Our Carrier Connections program features a different X-linked condition each month to increase awareness of X-linked conditions and how they impact the lives of women and girls.This month, we are featuring choroideremia (CHM). CHM is an X-linked disorder caused by mutations in the gene CHM, which produces a protein that plays a critical role in the cell's ability to transport proteins and organelles within and outside of the cell. When this gene is dysfunctional, the cell can no longer support this protein escort ability, resulting in premature cell death, primarily in the eyes. Typically, this condition is characterized by progressive vision loss. These symptoms may begin at any age, but tend to onset between childhood and adulthood. Females carriers of CHM have been proven to experience a spectrum of symptoms, ranging from mild to severe retinal degeneration. Our guest today is Michelle. She is a mom of two incredible boys with CHM, doing whatever she can to find a treatment. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠⁠⁠.

*This month in partnership with Choroideremia Research Foundation**Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls.This month, we are featuring choroideremia (CHM). CHM is an X-linked disorder caused by mutations in the gene, CHM, which produces a protein that plays a critical role in the cell's ability to transport proteins and organelles within and outside of the cell. When this gene is dysfunctional, the cell can no longer support this protein escort ability, resulting in premature cell death primarily in the eyes. Typically, this condition is characterized by progressive vision loss. These symptoms may begin at any age, but tend to onset between childhood and adulthood. Females carriers of CHM have been proven to experience a spectrum of symptoms, ranging from mild to severe retinal degeneration. Today, we are featuring Dr. Sena Gocuk. Dr. Gocuk is an optometrist and postdoctoral research fellow specialising in inherited retinal diseases (IRD), with a particular focus on female carriers of X-linked IRDs. Her research explores the unique challenges female carriers face, from variability in disease expression to their underrepresentation in clinical trials. Dr. Gocuk has led innovative studies investigating retinal changes in female carriers, providing insights into emerging treatments such as gene therapy. She is an advocate for the inclusion of female carriers in research and treatment interventions, regularly sharing her findings to promote better care and access for this often-overlooked population.RESOURCES:Choroideremia Research FoundationRetinal Characteristics of Female Choroideremia Carriers: Multimodal Imaging, Microperimetry, and GeneticsLongitudinal assessment of female carriers of choroideremia using multimodal retinal imagingFemale carriers of X-linked inherited retinal diseases - Genetics, diagnosis, and potential therapiesCarrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠⁠.

On this episode of Biotech Hangout, Eric Schmidt, Bruce Booth, Yaron Werber, Tim Opler and Mike Yee begin the show discussing the highlights from the Jefferies Healthcare Conference before taking a look at public investor sentiment this week. The discussion turns to Atlas Venture's 2024 Year in Review and transitions into the emerging themes for 2025. The hosts also recap ACR Convergence including Amgen's Phase 3 data as well as data from cell therapies for SLE, SSC, IMNM. The group also discuss lipid readouts from Eli Lilly, Silence Therapeutics and NewAmsterdam Pharma. Other topics covered include the Incyte and Escient deal collapse, Kura Oncology's pact with Kyowa Kirin, and perspectives on Trump's nomination of Dr. Mehmet Oz to lead the CMS. This episode aired on November 22, 2024.

Jaehyuk (Jae) Choi, MD, Northwestern University Feinberg School of Medicine, Chicago, IL Recorded on January 7, 2025 Jaehyuk (Jae) Choi, MD Jack W. Graffin Associate Professor of Dermatology and of Biochemistry and Molecular Genetics Northwestern University Feinberg School of Medicine Chicago, IL In this episode, Dr. Jaehyuk Choi from Northwestern University, explores Cutaneous T-Cell Lymphoma (CTCL), covering key diagnostic tools, emerging therapies, and the importance of a multidisciplinary team approach. He delves into effective symptom management, including the psychosocial impact of pruritus, and discusses the latest ongoing research. Additionally, Dr. Choi highlights disparities in diagnosis, treatment access, and patient outcomes. Tune in today to gain valuable insights on this complex diagnosis! This episode is supported by Kyowa Kirin, Inc.

Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. From time to time we also feature topics important to the X-linked community. This month, we are highlighting the topic of discussing health history with your family and more specifically disclosing an X-linked diagnosis. Our guests for this episode are Savannah and Alexis. Savannah and her son, Titus, have ALD and were diagnosed through newborn screening in 2021. The Merrill's are a military family currently stationed in Virginia. Due to ALD, they pursued IVF and are expecting an ALD-free baby girl in April of 2025. Alexis is a two boys under two mom! Her youngest son was picked up on Florida's newborn screen in 2023 with adrenoleukodystrophy (ALD). Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠⁠⁠⁠.

This episode features an interview with Britt Byers, Executive Vice President of Human Resources North America at Kyowa Kirin. With three decades of experience, Britt has held senior roles in Pharmaceuticals like Sanofi and Novartis, as well as in financial services at Bank of America. She has deep expertise in R&D and global functions as well as Shared Service leadership with oversight for HR Operations.In this episode, Shawn and Britt discuss the importance of authentic and inclusive leadership, strategies for easing employee anxieties, and the power of showing kindness in the workplace.-------------------“Before you react to something in a meeting or a conversation, or if someone's upset, just be there in the moment. You might have a difference of opinion, but you're just creating a space for the conversation. But it is on leaders, I think, to create the tone that you're not numb to what's happening. You can't not mention something. Otherwise, you just don't look genuine. You don't look like there's a leader you can trust who's just not acknowledging something that could be an awful environment.” – Britt Byers-------------------Episode Timestamps:*(02:54): Getting to know Britt*(14:34): The shift from financial services to pharmaceuticals*(18:10): The importance of meaningful work *(23:01): Leadership themes and enterprise leadership*(27:51): The importance of being kind in the workplace-------------------Links:Connect with Britt on LinkedInLearn more about Kyowa KirinFollow Kyowa Kirin on LinkedInConnect with Shawn on LinkedInCohesion PodcastAbout Simpplr

Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring creatine transported deficiency, or CTD. We are partnering with ⁠Association Xtraordinaire⁠ to bring you interviews and educational graphics on our social media pages. Our guests for this episode is Carole Chehowah, Vice President of Association Xtraordinaire. She is a mom of a 34-year-old daughter with CTD who diagnosed after 16 years after years of wondering. Carole is fighting for girls' recognition in X mutation syndromes. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠⁠⁠.

Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring creatine transported deficiency, or CTD. We are partnering with Association Xtraordinaire to bring you interviews and educational graphics on our social media pages. Our guests for this episode are Bonnie, an 18-year-old tennis player living with CTD and her mother, Andrea. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠⁠.

Roberto Novoa, MD, FAAD interviewed by Olayemi Sokumbi, MD, FAAD

**REPOST** Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring ornithine transcarbamylase (OTC) deficiency is an X-linked genetic disorder marked by a complete or partial lack of the enzyme ornithine transcarbamylase. This enzyme is critical to the urea cycle as it is tasked with converting ammonia, a byproduct from the breakdown of excess protein subunits in the body, to urea so that the body can excrete it through the urine. In individuals with OTC deficiency, this enzyme does not function properly, resulting in the accumulation of ammonia in the bloodstream. The presence of excess ammonia in the blood causes complications in the nervous system, resulting in a range of severe to mild symptoms. OTC deficiency can become evident at any age, but has the most extreme effects when it occurs in infants. Symptoms in females are widely variable, with some individuals showcasing more severe clinical manifestations than others, and some not having any reported symptoms. For example, while some females only report chronic headaches following protein consumption, other individuals have been found to experience acute liver failure, hyperammonemia, and other symptoms typically seen in affected males. Additionally, some females report worsening symptoms during pregnancy and require close monitoring of protein levels by a medical team to ensure a safe delivery. Even so, females are at a heightened risk of hyperammonemic crisis during pregnancy and should consult their physician before getting pregnant. OTC deficiency is an X-linked genetic disorder marked by a complete or partial lack of the enzyme ornithine transcarbamylase. This results in the accumulation of ammonia in the bloodstream and causes complications in the nervous system, resulting in a range of severe to mild symptoms. Approximately 20% of OTC females experience symptoms. Some females do not express excess ammonia levels until pregnancy. Females who do develop symptoms in childhood often experience headaches after protein ingestion. A study determined that girls with OTC deficiency may exhibit episodes of hyperammonemic encephalopathy, or ammonia buildup in the brain, and cognitive decline. Today, we are joined by Andrea Gropman, M.D., FAAP, FACMG, FANA. Dr. Gropman is the Director of Neurometabolic Translational Research Center for Experimental Neurotherapeutics St. Jude Children's Research Hospital. She is also is the principal investigator of the Urea Cycle Disorders Consortium (UCDC) and the UCDC imaging consortium and the deputy clinical director of the Mito EpiGen Program. Her research interest focuses on establishing biomarkers of neurological injury in patients with inborn errors of metabolism, such as urea cycle disorders and mitochondrial disorders, using specialized neuroimaging modalities and cell markers. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠⁠. OTC resources: ⁠https://checkammonia.com/⁠ ⁠https://www.ucdincommon.com/⁠ ⁠https://nucdf.org/

Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring ornithine transcarbamylase (OTC) deficiency is an X-linked genetic disorder marked by a complete or partial lack of the enzyme ornithine transcarbamylase. This enzyme is critical to the urea cycle as it is tasked with converting ammonia, a byproduct from the breakdown of excess protein subunits in the body, to urea so that the body can excrete it through the urine. In individuals with OTC deficiency, this enzyme does not function properly, resulting in the accumulation of ammonia in the bloodstream. The presence of excess ammonia in the blood causes complications in the nervous system, resulting in a range of severe to mild symptoms. OTC deficiency can become evident at any age, but has the most extreme effects when it occurs in infants. Symptoms in females are widely variable, with some individuals showcasing more severe clinical manifestations than others, and some not having any reported symptoms. For example, while some females only report chronic headaches following protein consumption, other individuals have been found to experience acute liver failure, hyperammonemia, and other symptoms typically seen in affected males. Additionally, some females report worsening symptoms during pregnancy and require close monitoring of protein levels by a medical team to ensure a safe delivery. Even so, females are at a heightened risk of hyperammonemic crisis during pregnancy and should consult their physician before getting pregnant. Today we are brining on Kristy McCracken, a urea cycle disorder advocate who is making rare extraordinary. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠. OTC resources: https://checkammonia.com/ https://www.ucdincommon.com/ https://nucdf.org/

*This month in partnership with ⁠⁠⁠⁠PPMD⁠⁠* Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring Duchenne muscular dystrophy (DMD). Duchenne is an X-linked disorder characterized by progressive muscle weakening and is one of the most common life-limiting genetic conditions diagnosed in childhood. It is caused by a mutation in the DMD gene, which typically functions to produce a protein critical for muscle structure and function known as dystrophin. In individuals with Duchenne, this protein is absent, leading to the degeneration of muscle fibers over time and progressive muscle weakening. Duchenne is a multi-systemic condition, which means it affects many parts of the body including the heart and respiratory muscles, contributing to cardiology and breathing-related complications. Females carriers are usually not affected with Duchenne or Becker because they make enough of the dystrophin protein. However, they can have some symptoms of Duchenne, such as changes to heart function, mild muscle weakness, fatigue, or cramping in their muscles. Rarely, a carrier has symptoms that are as severe as those of a male with muscular dystrophy. The term “manifesting carrier” is often used to describe a woman who has some of the symptoms of a male with Duchenne. Carrier females have an increased chance of changes to heart function. Because a small percentage of carriers may have serious heart concerns, it is important for all women who are carriers and women who are at risk to be carriers to have regular heart evaluations, beginning in their late teens/early adult years and, if normal, repeated every 3-5 years. Today, we are featuring Dr. Mena Scavina. Dr. Mena Scavina is Professor of Pediatrics and Neurology at Thomas Jefferson University and neurologist in the Division of Neurology at Nemours Children's Health, Delaware (retired). She completed her residency in Neurology at Thomas Jefferson University Hospital followed by an MDA Research Fellowship at the Children's Hospital of Philadelphia, and a clinical fellowship in neuromuscular diseases at Nemours. She was Director of the Neuromuscular Program at Nemours, Co-Director of the MDA Certified Center since 1997 and Director of the Parent Project Muscular Dystrophy Certified Duchenne Care Center since 2017 when the clinic was certified. Dr. Scavina is a consultant for Parent Project Muscular Dystrophy and has been involved with the Certified Duchenne Care Center (CDCC) program, certifying centers throughout the country. One of her interests is in the care of carriers of Duchenne and Becker muscular dystrophy and has been involved with the development of the Duchenne and Becker Carrier Clinic in conjunction with PPMD and cardiologists at the Penn Center for Inherited Cardiovascular Disease in Philadelphia. She is certified by the American Board of Psychiatry and Neurology and a member of the American Academy of Neurology. RESOURCES: PPMD Website and section on Carriers Decode Duchenne Information PPMD Registry information Penn Medicine Duchenne and Becker Carrier Clinic PPMD Lighthouse Workshops If individuals are interested in learning more about the Monthly Virtual Carrier Meeting, they can contact emily@parentprojectmd.org. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠.

*This month in partnership with ⁠⁠⁠PPMD⁠* Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring Duchenne muscular dystrophy (DMD). Duchenne is an X-linked disorder characterized by progressive muscle weakening and is one of the most common life-limiting genetic conditions diagnosed in childhood. It is caused by a mutation in the DMD gene, which typically functions to produce a protein critical for muscle structure and function known as dystrophin. In individuals with Duchenne, this protein is absent, leading to the degeneration of muscle fibers over time and progressive muscle weakening. Duchenne is a multi-systemic condition, which means it affects many parts of the body including the heart and respiratory muscles, contributing to cardiology and breathing-related complications. Females carriers are usually not affected with Duchenne or Becker because they make enough of the dystrophin protein. However, they can have some symptoms of Duchenne, such as changes to heart function, mild muscle weakness, fatigue, or cramping in their muscles. Rarely, a carrier has symptoms that are as severe as those of a male with muscular dystrophy. The term “manifesting carrier” is often used to describe a woman who has some of the symptoms of a male with Duchenne. Carrier females have an increased chance of changes to heart function. Because a small percentage of carriers may have serious heart concerns, it is important for all women who are carriers and women who are at risk to be carriers to have regular heart evaluations, beginning in their late teens/early adult years and, if normal, repeated every 3-5 years. Today we are chatting with Alexandra Wells. Alexandra is a 26-year-old Arizona native who enjoys country concerts, watching college football, and doing DIY projects. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠.

*This month in partnership with ⁠⁠PPMD* Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring Duchenne muscular dystrophy (DMD). Duchenne is an X-linked disorder characterized by progressive muscle weakening and is one of the most common life-limiting genetic conditions diagnosed in childhood. It is caused by a mutation in the DMD gene, which typically functions to produce a protein critical for muscle structure and function known as dystrophin. In individuals with Duchenne, this protein is absent, leading to the degeneration of muscle fibers over time and progressive muscle weakening. Duchenne is a multi-systemic condition, which means it affects many parts of the body including the heart and respiratory muscles, contributing to cardiology and breathing-related complications. Females carriers are usually not affected with Duchenne or Becker because they make enough of the dystrophin protein. However, they can have some symptoms of Duchenne, such as changes to heart function, mild muscle weakness, fatigue, or cramping in their muscles. Rarely, a carrier has symptoms that are as severe as those of a male with muscular dystrophy. The term “manifesting carrier” is often used to describe a woman who has some of the symptoms of a male with Duchenne. Carrier females have an increased chance of changes to heart function. Because a small percentage of carriers may have serious heart concerns, it is important for all women who are carriers and women who are at risk to be carriers to have regular heart evaluations, beginning in their late teens/early adult years and, if normal, repeated every 3-5 years. Today we are chatting with Trina Stelly. Trina is a wife and mom of three and and lives in south Louisiana. In her free time, she enjoy spending time taking care of her little farmstead and all of the miniature animals. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠rememberthegirls.org⁠⁠⁠.

*This month in partnership with ⁠⁠ALD Connect⁠⁠* Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring adrenoleukodystrophy (ALD). ALD is a X-linked genetic disorder characterized by the deterioration of myelin, a fatty substance that serves to insulate the nerves of the brain and spinal cord. In individuals with ALD, this loss of myelin, also known as demyelination, occurs due to an issue with the gene ABCD1. Under normal conditions, this gene produces a protein essential for breaking down a class of fatty acids known as very long-chain fatty acids, or VLCFAs. When ABCD1 malfunctions, however, VLCFAs build up in the body, creating inflammation that causes myelin to deteriorate. Females with ALD have been known to present symptoms similar to males with AMN, with the exception being that these symptoms tend to manifest later in life. Rarely do females develop the cerebral version of the disease or adrenal insufficiency. The following symptoms have been documented in females with ALD: Bowel and bladder dysfunction Tingling in the hands and feet (peripheral neuropathy) Excessive muscle tone Sexual dysfunction Poor bowel and bladder control Abnormal muscle reflexes Gait disorders Walking and balance issues Today we are chatting with Dr. Florian Eichler. Dr. Eicher is an Associate Professor of Neurology at Harvard Medical School. His research focus is on the genetics of peroxisomal disorders, lipid metabolism, and spatial aspects of nuclear magnetic resonance spectroscopy. As director of the leukodystrophy clinic at the Massachusetts General Hospital he sees patients with a variety of white matter disorders. He currently holds several NIH awards funding studies to analyze metabolic changes seen in the brain by MR measures and to determine the neurotoxicity of newly discovered atypical sphingolipids. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠rememberthegirls.org⁠⁠⁠.

*This month in partnership with ⁠ALD Connect⁠* Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring adrenoleukodystrophy (ALD). ALD is a X-linked genetic disorder characterized by the deterioration of myelin, a fatty substance that serves to insulate the nerves of the brain and spinal cord. In individuals with ALD, this loss of myelin, also known as demyelination, occurs due to an issue with the gene ABCD1. Under normal conditions, this gene produces a protein essential for breaking down a class of fatty acids known as very long-chain fatty acids, or VLCFAs. When ABCD1 malfunctions, however, VLCFAs build up in the body, creating inflammation that causes myelin to deteriorate. Females with ALD have been known to present symptoms similar to males with AMN, with the exception being that these symptoms tend to manifest later in life. Rarely do females develop the cerebral version of the disease or adrenal insufficiency. The following symptoms have been documented in females with ALD: Bowel and bladder dysfunction Tingling in the hands and feet (peripheral neuropathy) Excessive muscle tone Sexual dysfunction Poor bowel and bladder control Abnormal muscle reflexes Gait disorders Walking and balance issues Today we are chatting with Riham. Riham, a 45-year-old Canadian mother of three wonderful boys—the eldest two are ALD heroes—has transitioned from a career as a civil engineer to a dedicated advocate for rare diseases. She enjoys planting flowers and is passionate about researching rare diseases. As a co-founder of "ALD Hope", Canada's first nonprofit organization for ALD, she assists individuals with rare diagnoses in both English and Arabic. If she spoke more languages, she would undoubtedly offer her support on a global scale. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠rememberthegirls.org⁠⁠.

This episode, Matthew and Heather chat with Paul Testa, an Executive Vice President of Kyowa Kirin, a pharma group that develops innovative specialty drugs to raise the health and well being of people across the globe. Paul joins us to discuss Kyowa Kirin's expansion and economic impact in North Carolina. We also dive into treatment trends for rare diseases as well as the current landscape of supply and demand of medicine post-covid. Tune in now!

*This month in partnership with ALD Connect* Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring adrenoleukodystrophy (ALD). ALD is a X-linked genetic disorder characterized by the deterioration of myelin, a fatty substance that serves to insulate the nerves of the brain and spinal cord. In individuals with ALD, this loss of myelin, also known as demyelination, occurs due to an issue with the gene ABCD1. Under normal conditions, this gene produces a protein essential for breaking down a class of fatty acids known as very long-chain fatty acids, or VLCFAs. When ABCD1 malfunctions, however, VLCFAs build up in the body, creating inflammation that causes myelin to deteriorate. Females with ALD have been known to present symptoms similar to males with AMN, with the exception being that these symptoms tend to manifest later in life. Rarely do females develop the cerebral version of the disease or adrenal insufficiency. The following symptoms have been documented in females with ALD: Bowel and bladder dysfunction Tingling in the hands and feet (peripheral neuropathy) Excessive muscle tone Sexual dysfunction Poor bowel and bladder control Abnormal muscle reflexes Gait disorders Walking and balance issues Today we are chatting with Jana. Jana is from Orange County, California where she enjoys spending time with family and friends, especially her husband Rick and daughter, Olivia. She's fighting ALD with everything she has. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠rememberthegirls.org⁠.

Our Carrier Connections program features a different X-linked condition or community topic each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are highlighting the topic of discussing X-linked inheritance and carrier status with your daughter(s). Aaron Gardner joined us on this episode to share his story and experience navigating this thus far. Aaron Gardner is a 38-year-old man from Washington state who was diagnosed with adrenoleukodystrophy (ALD) nine years ago. He enjoys cooking, playing wheelchair basketball, and spending time with his wife and two kids (one of whom also has the gene mutation which causes ALD). Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠.

Our Carrier Connections program features a different X-linked condition or community topic each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are highlighting the topic of discussing X-linked inheritance and carrier status with your daughter(s). Isabella & Beth, a mother-daughter duo who are both manifesting carriers of myotubular myopathy, have joined us on an episode to share their lived experience. Beth Bauer is mom to Isabella and she believes "knowledge is power and with a positive attitude anything can be accomplished!" Isabella believes "we may have to look at things differently or do things differently, but we can still have a great life!" Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠.

Our Carrier Connections program features a different X-linked condition or community topic each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are highlighting the topic of discussing X-linked inheritance and carrier status with your daughter(s). Elsa and Ann have joined us on an episode to share their lived experience. Elsa is a student, lover of books, and founder of the Red Ink Project. Ann is a mother, advocate, writer, and English professor. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠rememberthegirls.org⁠⁠⁠.

In this week's episode, Danie du Plessis, Executive Vice President, Pharmaceutical Operations, Kyowa Kirin, sits down with the EMJ GOLD team to share the value that patient partnerships can hold in the industry. Danie explores how patient partnerships have evolved over the years, what companies should be doing to get them to their best potential, the value of learning from others and much more. A little more on EMJ GOLD's guest… After completing his medical studies and a Master's Degree in Clinical Pharmacology in South Africa, Danie spent three years working in a primary care setting including his own private practice. He joined the pharmaceutical industry in 1993, worked in medical affairs, marketing and sales, pharmacovigilance and clinical operations. He has held roles such as including Senior Vice President and Head of Worldwide Medical Affairs at GlaxoSmithKline. Danie joined Kyowa Kirin International in August 2019 as head of Medical Affairs and has been Executive Vice President Pharmaceutical Operations since April 2024.  He is also a Director and Chair of the Global non-profit Medical Affairs Professional Society (MAPS) Board, Fellow of the Faculty of Pharmaceutical Medicine in the UK, visiting senior lecturer at King's College, London and an affiliate member of the Association for Professional Executive Coaching and Supervision (APECS). 

Our Carrier Connections program features a different X-linked condition or community topic each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are highlighting the topic of discussing X-linked inheritance and carrier status with your daughter(s). Today we are chatting with Ali Conn. Ali is a pediatric genetic counselor at Rush University Medical Center in Chicago (all views expressed are her own). She has an undergraduate degree in biomedical engineering, but confirmed that her teenage love for Punnett squares actually was her calling when she graduated from genetic counseling school in 2022. She loves working with pediatric patients and their families, seeking diagnoses for adults with autism, LGBTQ+ advocacy, and will probably show you pictures of her dogs. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠rememberthegirls.org⁠⁠.

*This month in partnership with ⁠XLH Network⁠* Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring X-linked hypophosphatemia (XLH). XLH is a genetic disease caused by mutations in the PHEX gene. These mutations cause bone cells to secrete increased concentrations of a hormone responsible for inducing phosphate excretion in the kidneys, ultimately resulting in a deficiency of phosphate in the body. This can cause widespread issues throughout the body as phosphate plays an essential role in the proper functioning of bones and teeth. Most cases of XLH first manifest in childhood, with the presentation of bowed legs appearing as a prominent symptom. XLH impacts females in virtually the same way it impacts males. Today we are chatting with Elizabeth Olear. Elizabeth Olear is the Senior Clinical Research Associate at the Yale Center for XLH in the division of Pediatric Endocrinology at Yale School of Medicine. She leads a team conducting clinical trials in rare bone metabolism and research involving long-term sequelae of these disorders. She has been at Yale for over 17 years and has been actively involved in the research efforts on X-linked hypophosphatemia. Elizabeth has a strong commitment to advocacy and education for patients in the rare disease community. She currently serves on the Board of Directors for The XLH Network, Inc. and is a founding member of The International XLH Alliance. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠rememberthegirls.org⁠.

In this CME episode, Dr. Andrew Cutler interviews Dr. Laxman Bahroo about the current landscape of tardive dyskinesia, including screening, diagnosis, and treatment. Practical strategies based on the latest data are reviewed at length, particularly for long-term management of tardive dyskinesia.  Target Audience: This activity has been developed for the healthcare team or individual prescriber specializing in mental health. All other healthcare team members interested in psychopharmacology are welcome for advanced study. Learning Objectives: After completing this educational activity, you should be better able to: Consider the individual patient profile and symptoms when determining a treatment plan for patients with tardive dyskinesia Integrate latest safety, efficacy, and tolerability data of VMAT2 inhibitors into decision-making processes when considering treatment options for tardive dyskinesia Evaluate practical strategies for patient follow-up and long-term VMAT2 inhibitor treatment Evaluate and integrate recent updates and advancements regarding evidence-based treatment strategies for tardive dyskinesia Accreditation: In support of improving patient care, Neuroscience Education Institute (NEI) is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team. Activity Overview: This activity is available with audio and is best supported via a computer or device with current versions of the following browsers: Mozilla Firefox, Google Chrome, or Safari. A PDF reader is required for print publications. A post-test score of 70% or higher is required to receive CME/CE credit. Estimated Time to Complete: 1 hour Released: June 26, 2024*   Expiration: June 25, 2027 *NEI maintains a record of participation for six (6) years. CME/CE Credits and Certificate Instructions: After listening to the podcast, to take the optional posttest and receive CME/CE credit, click: https://nei.global/POD24-02-TD Credit Designations: The following are being offered for this activity: Physician: ACCME AMA PRA Category 1 Credits™ NEI designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity Nurse: ANCC contact hours NEI designates this Enduring Material for a maximum of 1.0 ANCC contact hours Nurse Practitioner: ACCME AMA PRA Category 1 Credit™ American Academy of Nurse Practitioners National Certification Program accepts AMA PRA Category 1 Credit™ from organizations accredited by the ACCME. The content in this activity pertaining to pharmacology is worth 1.0 continuing education hour of pharmacotherapeutics. Pharmacy: ACPE application-based contact hours This internet enduring, knowledge-based activity has been approved for a maximum of 1.0 contact hour (.10 CEUs). The official record of credit will be in the CPE Monitor system. Following ACPE Policy, NEI must transmit your claim to CPE Monitor within 60 days from the date you complete this CPE activity and is unable to report your claimed credit after this 60-day period. Physician Associate/Assistant: AAPA Category 1 CME credits NEI has been authorized by the American Academy of PAs (AAPA) to award AAPA Category 1 CME credit for activities planned in accordance with the AAPA CME Criteria. This internet enduring activity is designated for 1.0 AAPA Category 1 credit. Approval is valid until June 25, 2027. PAs should only claim credit commensurate with the extent of their participation. Psychology: APA CE credits Continuing Education (CE) credits for psychologists are provided through the co-sponsorship of the American Psychological Association (APA) Office of Continuing Education in Psychology (CEP). The APA CEP Office maintains responsibility for the content of the programs. Social Work: ASWB-ACE CE credits As a Jointly Accredited Organization, NEI is approved to offer social work continuing education by the Association of Social Work Boards (ASWB) Approved Continuing Education (ACE) program. Organizations, not individual courses, are approved under this program. Regulatory boards are the final authority on courses accepted for continuing education credit. Social workers completing this internet enduring course receive 1.0 general continuing education credits. Non-Physician Member of the Healthcare Team: Certificate of Participation NEI awards hours of participation (consistent with the designated number of AMA PRA Category 1 Credit(s)™) to a participant who successfully completes this educational activity. Interprofessional Continuing Education: IPCE credit for learning and change This activity was planned by and for the healthcare team, and learners will receive 1 Interprofessional Continuing Education (IPCE) credit for learning and change. Peer Review: The content was peer-reviewed by an MD, MPH specializing in forensics, psychosis, schizophrenia, mood disorders, anxiety, cognitive disorders — to ensure the scientific accuracy and medical relevance of information presented and its independence from commercial bias. NEI takes responsibility for the content, quality, and scientific integrity of this CME/CE activity. Disclosures: All individuals in a position to influence or control content are required to disclose any relevant financial relationships. Faculty Author / Presenter Andrew J. Cutler, MD Clinical Associate Professor, Department of Psychiatry and Behavioral Sciences, Norton College of Medicine, State University of New York Upstate Medical University, Syracuse, NY Chief Medical Officer, Neuroscience Education Institute, Malvern, PA Consultant/Advisor: AbbVie, Acadia, Alfasigma, Alkermes, Axsome, Biogen, BioXcel, Boehringer Ingelheim, Brii Biosciences, Cerevel, Corium, Delpor, Evolution Research, Idorsia, Intra-Cellular, Ironshore, Janssen, Jazz, Karuna, Lundbeck, LivaNova, Luye, MapLight Therapeutics, Neumora, Neurocrine, NeuroSigma, Noven, Otsuka, Relmada, Reviva, Sage Therapeutics, Sumitomo (Sunovion), Supernus, Takeda, Teva, Tris Pharma, VistaGen Therapeutics Speakers Bureau: AbbVie, Acadia, Alfasigma, Alkermes, Axsome, BioXcel, Corium, Idorsia, Intra-Cellular, Ironshore, Janssen, Lundbeck, Neurocrine, Noven, Otsuka, Sumitomot (Sunovion), Supernus, Takeda, Teva, Tris Pharma, Vanda Data Safety Monitoring Board (DSMB): COMPASS Pathways, Freedom Biosciences Faculty Author / Presenter Laxman B. Bahroo, DO, MS, FAAN Professor and Residency Program Director, Department of Neurology, MedStar Georgetown University Hospital, Washington, DC Consultant/Advisor: AbbVie, Amneal, Ipsen, Kyowa Kirin, Merz, Neurocrine, Revance, Supernus Speakers Bureau: AbbVie, Acadia, Acorda, Amneal, Ipsen, Kyowa Kirin, Merz, Neurocrine, Supernus, Teva The remaining Planning Committee members, Content Editors, Peer Reviewer, and NEI planners/staff have no financial relationships to disclose. NEI planners and staff include Caroline O'Brien, Meghan Grady, Brielle Calleo, and Andrea Zimmerman, EdD. Disclosure of Off-Label Use: This educational activity may include discussion of unlabeled and/or investigational uses of agents that are not currently labeled for such use by the FDA. Please consult the product prescribing information for full disclosure of labeled uses. Cultural Linguistic Competency and Implicit Bias: A variety of resources addressing cultural and linguistic competencies and strategies for understanding and reducing implicit bias can be found in this handout—download me. Accessibility Statement Contact Us: For questions regarding this educational activity, or to cancel your account, please email customerservice@neiglobal.com. Support: This activity is supported by an unrestricted educational grant from Neurocrine Biosciences.

*This month in partnership with XLH Network* Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring X-linked hypophosphatemia (XLH). XLH is a genetic disease caused by mutations in the PHEX gene. These mutations cause bone cells to secrete increased concentrations of a hormone responsible for inducing phosphate excretion in the kidneys, ultimately resulting in a deficiency of phosphate in the body. This can cause widespread issues throughout the body as phosphate plays an essential role in the proper functioning of bones and teeth. Most cases of XLH first manifest in childhood, with the presentation of bowed legs appearing as a prominent symptom. XLH impacts females in virtually the same way it impacts males. Today we are chatting with Shannon Sharp. Shannon resides in Troy, Alabama with her husband and grandbaby. She has known of her condition essentially her entire life as her father and grandmother were both patients with XLH, though originally diagnosed with vitamin-D resistant rickets prior to genetic testing. Shannons first symptom of XLH was severe bowing of the legs along with fatigue and joint and bone pain. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out rememberthegirls.org.

Each month, our Carrier Connections program features a different X-linked condition with the goal to increase awareness and education of X-linked conditions and how they impact females. This month, we are featuring X-linked hypophosphatemia (XLH). XLH is a genetic disease caused by mutations in the PHEX gene, which cause bone cells to secrete increased concentrations of a hormone responsible for inducing phosphate excretion in the kidneys, ultimately resulting in a deficiency of phosphate in the body. This can cause widespread issues throughout the body as phosphate plays an essential role in the proper functioning of bones and teeth. Most cases of XLH first manifest in childhood, with the presentation of bowed legs appearing as a prominent symptom.  Today, we are bringing on Lauren Walrath, Vice President of Public Affairs at Kyowa Kirin North America. Lauren joined Kyowa Kirin in 2019 and has been responsible for building and leading the region's Public Affairs team, including its communications and advocacy capabilities, to support the company's culture, reputation and work with patient communities. She is a key member of the company's North America Executive Committee and Global Corporate Communications leadership team. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out rememberthegirls.org.

Qu'est-ce que l'ostéomalacie ? Quelles sont les causes rares d'ostéomalacies ? Quels sont les signes d'appel clinique et biologique de l'ostéomalacie ? Quand mettre en place un traitement ?   Le Dr Guillaume Couture, rhumatologue au CHU de Toulouse et praticien au Centre de référence des pathologies phospho-calciques de la filière OSCAR, répond à vos questions.   Invité : Dr Guillaume Couture https://www.chu-toulouse.fr/-presentation-du-centre-de,1591-  Le Dr Guillaume Couture déclare des interventions ponctuelles et une activité d'expertise ponctuelle pour Kyowa Kirin.   L'équipe :
 Comité scientifique : Pr Jérémie Sellam, Pr Thao Pham, Dr Catherine Beauvais, Dr Véronique Gaud-Listrat, Dr Céline Vidal, Dr Sophie Hecquet Animation :  Pyramidale Communication Production : Pyramidale Communication Soutien institutionnel : Pfizer   Crédits : Pyramidale Communication, Sonacom

*This month in partnership with XLH Network* Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring X-linked hypophosphatemia (XLH). XLH is a genetic disease caused by mutations in the PHEX gene. These mutations cause bone cells to secrete increased concentrations of a hormone responsible for inducing phosphate excretion in the kidneys, ultimately resulting in a deficiency of phosphate in the body. This can cause widespread issues throughout the body as phosphate plays an essential role in the proper functioning of bones and teeth. Most cases of XLH first manifest in childhood, with the presentation of bowed legs appearing as a prominent symptom. XLH impacts females in virtually the same way it impacts males. Today we are bring on Sunindiya. Sunindiya lives outside of Boston, MA. She is the Executive Vice President for a nonprofit organization called Roca, where she manages programs for young women and mothers impacted by trauma and violence. Her experience living with XLH helped her find her voice to advocate for others who are often unseen and unheard both through her work and through The XLH Network. Sunindiya likes to spend time with family, read, and swim.  Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out rememberthegirls.org.

Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring myotubular myopathy (MTM). MTM is an X-linked genetic disorder characterized by moderate to severe muscle weakness and decreased muscle tone. Manifesting female carriers of an MTM1 mutation express a range of symptoms that vary in severity, but the following describe the general pattern seen across studies: Muscle weakness in upper and lower limbs  Muscle atrophy  Dyspnea  Muscle fatigue Cramping Calf enlargement  Wheelchair dependence Intolerance to physical activity  Contractures  Choking while eating  Vocal changes Today we are bring on Meghan Mustane to discuss her experience of being diagnosed a carrier with MTM. Meghan is an experienced communications professional working in Pharma and is currently on the board for Remember The Girls. She is a carrier of Myotubular Myopathy and always knew there was a chance she was a carrier from her mothers own journey in family planning and child loss. Meghan is a mother of two girls, age 14 and 9 and knows one day they will require testing to find out their own carrier status with the condition. The ethics of when to talk to your daughter and when to find out your children's own carrier journey is always a debated topic in our community and one that is very personal and also very grey. Meghan is passionate about raising awareness for rare diseases and we appreciate her continued efforts and support inside Remember The Girls. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out rememberthegirls.org.

Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring myotubular myopathy (MTM). MTM is an X-linked genetic disorder characterized by moderate to severe muscle weakness and decreased muscle tone. Manifesting female carriers of an MTM1 mutation express a range of symptoms that vary in severity, but the following describe the general pattern seen across studies: Muscle weakness in upper and lower limbs Muscle atrophy Dyspnea Muscle fatigue Cramping Calf enlargement Wheelchair dependence Intolerance to physical activity Contractures Choking while eating Vocal changes Today we are bring on Kim Engst to discuss her experience being diagnosed with MTM. Kim is a manifesting carrier of MTM. She has a daughter and a son who passed away from MTM. She is a retired Veterinary Technician of 40 years, a career she loved! She is a lizard mom, I have 3 crested geckos: Emilio, Flora and Fauna. She is a voracious reader and enjoy doing paint by numbers kits (great for mental health and keeping her hands moving!) Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠⁠.

Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring myotubular myopathy (MTM). MTM is an X-linked genetic disorder characterized by moderate to severe muscle weakness and decreased muscle tone. Manifesting female carriers of an MTM1 mutation express a range of symptoms that vary in severity, but the following describe the general pattern seen across studies: Muscle weakness in upper and lower limbs Muscle atrophy Dyspnea Muscle fatigue Cramping Calf enlargement Wheelchair dependence Intolerance to physical activity Contractures Choking while eating Vocal changes Today, we are interviewing Jessica Stanley, a female patient with myotubular myopathy. Jessica was mis-diagnosed multiple times and ultimately found out her MTM diagnosis through genetic testing. She is currently pregnant with twin baby girls and has been contemplating testing for their carrier status in utero. Women patients and carriers of x-linked conditions not only find themselves with physical symptoms of their condition but also the mental load of making these tough decisions can be daunting. Jessica is a beautiful example of how community and staying positive can ultimately help bring women together, sharing their journey with each other. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠.

*SOLIDARITY EPISODE* This month in partnership with ⁠Fabry Support & Information Group⁠! Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring Fabry disease. Fabry Disease is a rare X-linked disorder caused by a mutation in the gene encoding for the α-galactosidase A (α-Gal A) enzyme, or GLA gene, which typically functions to produce a protein required for the breakdown of lipids in cells. Females tend to have a variable course of Fabry disease with some experiencing severe symptoms while others experience less severe symptoms than males or none at all. However, recent research has determined that the most common symptoms in females affected by Fabry disease are neuropathic pain, angiokeratoma (clusters of small, dark red spots), proteinuria (high levels of protein in urine), buildup in the cornea of the eyes, and cardiac disease. Additionally, 10% of females experience renal failure and need dialysis. Depression is also reported to be another manifestation of Fabry disease with 22% of females experiencing depressive symptoms. Aside from these manifestations, more recent research has discovered females with Fabry disease tend to experience: exhaustion, exercise intolerance, decreased oxygen intake, gastroenterological disturbances, infertility, loss of libido, increased risk of headaches, and severity of Fabry-related symptoms in pregnancy. Today, we are bringing on Taylor and her mother, Andrea. Taylor is a young adult living with Fabry disease. She and Andrea discuss the challenges they faced getting Taylor's symptoms taken seriously, how their family supports Taylor through the trials and tribulations brought about by Fabry, and why they advocate. Find them here: Taylor's Instagram - @_friendswithfabry_ Andrea's email - andiemick@aol.com Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠⁠⁠⁠⁠.

This month in partnership with ⁠⁠Fabry Support & Information Group! This month, we are featuring Fabry disease. Fabry Disease is a rare X-linked disorder caused by a mutation in the gene encoding for the α-galactosidase A (α-Gal A) enzyme, or GLA gene, which typically functions to produce a protein required for the breakdown of lipids in cells. Females tend to have a variable course of Fabry disease with some experiencing severe symptoms while others experience less severe symptoms than males or none at all. However, recent research has determined that the most common symptoms in females affected by Fabry disease are neuropathic pain, angiokeratoma (clusters of small, dark red spots), proteinuria (high levels of protein in urine), buildup in the cornea of the eyes, and cardiac disease. Additionally, 10% of females experience renal failure and need dialysis. Depression is also reported to be another manifestation of Fabry disease with 22% of females experiencing depressive symptoms. Aside from these manifestations, more recent research has discovered females with Fabry disease tend to experience: exhaustion, exercise intolerance, decreased oxygen intake, gastroenterological disturbances, infertility, loss of libido, increased risk of headaches, and severity of Fabry-related symptoms in pregnancy. Today, we are bringing on Lisa Berry. Lisa Berry graduated from the Brandeis University Genetic Counseling program in Waltham, MA. She has worked as a prenatal counselor in New York City, a lab coordinator for Genzyme Genetics and a study coordinator at Tufts/New England Medical Center. In 2008, she became a genetic counselor with the Rare Genetic Disease Program at Cincinnati Children's Hospital. It was here that she was able to start working with individuals and families whose lives have been impacted by lysosomal storage disorders. In addition to providing genetic counseling for families, she is on the Ohio Newborn Screening Advisory Council and is a member of the Board of Directors for the National MPS Society. Her main roles are treatment/care coordination, advocacy and working on clinical trials for various LSDs. Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠⁠⁠⁠.

Hosted by Jalilah Gibson and Caz Hanrahan, our new XLR8 podcast series delves into answering your most common pain points and questions as you progress through your digital transformation journey in the pharma world. We'll be bringing you real-life stories and experiences from distinguished guests who have delivered customer and business value through the use of digital, starting by designing around the customer. In our first episode, Jalilah and Caz are joined by Andrew Peryer, former Head of Digital for Established Medicines at Kyowa Kirin. Andrew is a highly experienced senior leader with a demonstrated history of working in pharma marketing and business development. He's passionate about applying technology, customer-first thinking and data to healthcare problems through collaborative network models. In this episode, Andrew will explore: - Embracing a customer-first mindset - Transitioning from a product provider for your customer to a trusted partner - Understanding how experiences beyond our industry shape customer expectations - Overcoming challenges in reshaping your go-to-market strategy - Implementing an iterative approach in your digital transformation - Strategic prioritisation and investment strategies for success Listen to the full episode to accelerate your digital journey from theory to delivery. If you have any questions/or comments feel free to reach out to Jalilah or Caz via their LinkedIn here: Jalilah - https://www.linkedin.com/in/jalilah/ Caz - https://www.linkedin.com/in/catherine-caz-hanrahan-27455122/. Thank you

*SOLIDARITY EPISODE* This month in partnership with Fabry Support & Information Group! Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. This month, we are featuring Fabry disease. Fabry Disease is a rare X-linked disorder caused by a mutation in the gene encoding for the α-galactosidase A (α-Gal A) enzyme, or GLA gene, which typically functions to produce a protein required for the breakdown of lipids in cells. Females tend to have a variable course of Fabry disease with some experiencing severe symptoms while others experience less severe symptoms than males or none at all. However, recent research has determined that the most common symptoms in females affected by Fabry disease are neuropathic pain, angiokeratoma (clusters of small, dark red spots), proteinuria (high levels of protein in urine), buildup in the cornea of the eyes, and cardiac disease. Additionally, 10% of females experience renal failure and need dialysis. Depression is also reported to be another manifestation of Fabry disease with 22% of females experiencing depressive symptoms. Aside from these manifestations, more recent research has discovered females with Fabry disease tend to experience: exhaustion, exercise intolerance, decreased oxygen intake, gastroenterological disturbances, infertility, loss of libido, increased risk of headaches, and severity of Fabry-related symptoms in pregnancy. Today, we are bringing on Sabina Kineen. Sabina is a rare disease patient, caregiver, and advocate with a deep passion for health equity, mental health, and patient engagement. Diagnosed with Fabry Disease in her teens, she has spent many years sharing how the diagnosis of an inherited disease can impact an entire family.  Sabina is also a strong proponent of health equity and strives to ensure that every patient has access to the care they need and deserve. Through her advocacy work, Sabina aims to raise awareness, promote understanding, and create meaningful change for rare disease patients and their families.  Carrier Connections is sponsored by Kyowa Kirin and Amgen. For more information about our organization, check out ⁠⁠⁠⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠⁠⁠⁠.

*SOLIDARITY EPISODE* This month in partnership with ⁠Alport Syndrome Foundation⁠! Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. Some of these X-linked conditions are part of a larger disease umbrella with other types of inheritance patterns. This month, we are featuring Alport syndrome. Alport syndrome is an inherited disorder caused by mutations, or changes, in the genes COL4A3, COL4A4, and COL4A5 that inhibit their ability to produce a protein called collagen IV. There are four main types of Alport syndrome characterized by their mode of inheritance; the X-linked version of this syndrome, X-linked Alport syndrome (XLAS), is the most commonly reported and brought on specifically by a defective COL4A5 gene. Other inheritance forms include autosomal recessive, autosomal dominant, and digenic. Alport syndrome is most frequently associated with symptoms of progressive kidney disease as well as bilateral sensorineural hearing loss and eye abnormalities. Today, we are bringing on Rachel. Rachel is a 24 year old with X-linked Alport syndrome who lives in Boston and is an aspiring genetic counselor. She is passionate about raising awareness for the carrier community and advocating for women's healthcare rights. She loves to play soccer, ski, and read. Carrier Connections is sponsored by Kyowa Kirin, Amgen, and Sanofi. For more information about our organization, check out ⁠⁠⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠⁠⁠.

This month in partnership with ⁠Alport Syndrome Foundation⁠! Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. Some of these X-linked conditions are part of a larger disease umbrella with other types of inheritance patterns. This month, we are featuring Alport syndrome. Alport syndrome is an inherited disorder caused by mutations, or changes, in the genes COL4A3, COL4A4, and COL4A5 that inhibit their ability to produce a protein called collagen IV. There are four main types of Alport syndrome characterized by their mode of inheritance; the X-linked version of this syndrome, X-linked Alport syndrome (XLAS), is the most commonly reported and brought on specifically by a defective COL4A5 gene. Other inheritance forms include autosomal recessive, autosomal dominant, and digenic. Alport syndrome is most frequently associated with symptoms of progressive kidney disease as well as bilateral sensorineural hearing loss and eye abnormalities. Today, we are bringing on Jennie Feiger. Although she has no accent to reveal her origins, Jennie grew up in Brooklyn, New York.  She attended Wesleyan University for her undergraduate degree, and then earned two masters degrees from the University of California at Berkeley, in Biochemistry and Genetic Counseling. She has worked in genetic research, genetic counseling, and teaching at the college and graduate level.   As a genetic counselor, she focused on adult onset conditions, including cancer and dementia.  She has presented her findings at national meetings, as well as founded support groups for families impacted by adult-onset conditions and is active in training graduate students in genetic counseling.   As a physician assistant program, she has worked in internal medicine, geriatrics and gastroenterology, and now nephrology.  Jennie enjoys getting to know her patients deeply and takes pride in providing comprehensive care, including genetic counseling.  She lives in Boulder with her husband and is recently an “empty nester” with her two young sons in college.  She loves to garden, cycle and cross country ski. Carrier Connections is sponsored by Kyowa Kirin, Amgen, and Sanofi. For more information about our organization, check out ⁠⁠⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠⁠⁠. Novel therapies for Alport syndrome: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9081811/

*SOLIDARITY EPISODE* This month in partnership with Alport Syndrome Foundation! Our Carrier Connections program features a different X-linked condition each month with the goal to increase awareness of X-linked conditions and how they impact the lives of women and girls. Some of these X-linked conditions are part of a larger disease umbrella with other types of inheritance patterns. This month, we are featuring Alport syndrome. Alport syndrome is an inherited disorder caused by mutations, or changes, in the genes COL4A3, COL4A4, and COL4A5 that inhibit their ability to produce a protein called collagen IV. There are four main types of Alport syndrome characterized by their mode of inheritance; the X-linked version of this syndrome, X-linked Alport syndrome (XLAS), is the most commonly reported and brought on specifically by a defective COL4A5 gene. Other inheritance forms include autosomal recessive, autosomal dominant, and digenic. Alport syndrome is most frequently associated with symptoms of progressive kidney disease as well as bilateral sensorineural hearing loss and eye abnormalities. Today, we are bringing on December West. December has been married to Tarik West for 19 years. They have five children Alexis, Obed, Xyia, Nhalani, and Othniel. They reside in Akron, Ohio. Nhalani, who is 12-year-old now, was diagnosed with autosomal recessive Alport syndrome at the age of 6. Nhalani was misdiagnosed several times. Several years later, after a failed hearing screening and diagnosed with bilateral sensorineural hearing loss, Nhalani was referred to a geneticist who properly diagnosed her with autosomal recessive Alport syndrome. Carrier Connections is sponsored by Kyowa Kirin, Amgen, and Sanofi. For more information about our organization, check out ⁠⁠⁠⁠⁠⁠rememberthegirls.org⁠⁠⁠⁠⁠⁠.

Good morning from Pharma and Biotech daily, the podcast bringing you crucial updates from the Pharma and Biotech world. Today's episode covers several significant developments and deals in the industry.AstraZeneca is on the verge of acquiring vaccine maker Icosavax for up to $1.1 billion. This move underscores AstraZeneca's dedication to vaccine development, particularly as it gains a combination vaccine targeting respiratory viruses. This acquisition is timely, considering the looming expiration of patents for several major medicines, a challenge prompting big pharma to explore new markets.In a notable development, Bristol Myers Squibb (BMS) has acquired rights to develop and commercialize Systimmune's antibody-drug conjugate (ADC) for various cancers, including lung and breast cancer. The deal, potentially worth $8.4 billion, highlights the growing interest in ADCs, a promising class of cancer treatments.Sanofi has terminated its licensing agreement with Maze Therapeutics for a Pompe disease drug following challenges from the US Federal Trade Commission (FTC). The decision to end this deal reflects the heightened regulatory scrutiny within the industry.Reiterating its focus on vaccines, AstraZeneca has also acquired Icosavax in a deal potentially worth $1.1 billion. This acquisition enables AstraZeneca to enter the market for respiratory syncytial virus vaccines, demonstrating its commitment to expanding its vaccine portfolio.In a major industry move, Pfizer's $43 billion acquisition of Seagen has overcome regulatory barriers and is set to conclude. This deal, the largest in recent years, will significantly enhance Pfizer's presence in the ADC market, a trend indicative of the current wave of large-scale acquisitions in pharma.Sino Biological, a biotech firm, has made strides in gene therapy by developing platforms for multi-pass transmembrane protein products. These advancements underscore the industry's progress in drug manufacturing technologies.Employee engagement is gaining importance in the sector, as illustrated by Kyowa Kirin's new program aimed at involving its workforce in shaping the company's future. This approach reflects a broader understanding of the role of employee involvement in fostering innovation and success.Tome Biosciences has launched with a significant funding of $213 million, focusing on genomic medicines, particularly in precision oncology. This move highlights the ongoing advancements in gene therapy and personalized medicine.At the American Society of Hematology (ASH) annual meeting, BMS and 2seventy Bio presented promising survival data for their CAR-T therapy, Abecma, used in treating multiple myeloma. This data points to the potential of CAR-T therapy in treating complex diseases.Novartis' iptacopan showed positive results in a Phase III study for an ultra-rare kidney disease. Coming shortly after FDA approval for another indication, this demonstrates progress in treating rare diseases.Catalent is seeking to expand in the GLP-1 market, used in treating type 2 diabetes, amid a revenue downturn and staff cuts. This move reflects the competitive nature of the diabetes treatment market.The Centers for Medicare and Medicaid Services (CMS) are preparing to announce the 'maximum fair price' for drugs under the Inflation Reduction Act's negotiation program. This impending decision is poised to be a pivotal pharmaceutical trend in 2024, with significant political and economic implications, especially considering the upcoming presidential election.The year 2023 saw four historic FDA approvals, including a vaccine for RSV and a drug for Alzheimer's disease, showcasing ongoing progress in drug development and healthcare.Bryter's insights have been instrumental in pharma's success in marketing rare disease treatments, highlighting the vital role of data analytics and strategic insights in effective marketin

Jointly Provided by the American Academy of CME and CheckRare CE Support for this accredited continuing education activity has been made possible through an educational grant from Kyowa Kirin. Estimated time to complete: 0.50 hours Start date: November 30, 2023 End date: November 30, 2024 This 15-minute CME-accredited program, hosted by Aliya Khan, MD, Clinical Professor of Medicine at McMaster University, highlights the best practices to diagnose tumor induced osteomalacia (TIO) based on the recently published guidelines in the Journal of Internal Medicine. To earn credit, go to https://checkrare.com/learning/p-new-guidance-to-diagnose-and-treat-tumor-induced-osteomalacia-tio-2023-1/ Activity Faculty Aliya Khan MD, FRCPC, FACP, FACE, FASBMR Clinical Professor of Medicine Director, Calcium Disorders Clinic Director, Fellowship in Metabolic Bone Disease McMaster University Target Audience: This activity is designed to meet the educational needs of physicians specializing in neurology, orthopedics, internal medicine/general practice, rheumatology, endocrinology, pain management, and radiology, who may be involved in the care for individuals with TIO. Other healthcare providers may also take part. Learning Objectives: After participating in the activity, learners should be better able to • Describe the latest recommendations for diagnosing patients with TIO• Describe the latest recommendations for treating patients with TIO Accreditation and Credit Designation: In support of improving patient care, this activity has been planned and implemented by the American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is Jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team. Physicians American Academy of CME, Inc., designates this enduring material for a maximum of 0.25 AMA PRA Category 1 Credits. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Other members of the care team will receive a certificate of participation. Disclosure Statement: According to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated. Disclosure of relevant financial relationships are as follows: Faculty Educator Dr. Khan discloses the following relevant financial relationships with ineligible companies to disclose: • Advisory Board/Consultant: Amgen, Ascendis, Alexion • Grant/Research support: Ascendis, Alexion, Amolyt • Speakers Bureaus: Amgen, Ascendis, Alexion Planners for this activity have no relevant financial relationships with any ineligible companies. This activity will review off-label or investigational information. The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically and draw conclusions only after careful consideration of all available scientific information. Method of Participation: There are no fees to participate in the activity. Participants must review the activity information, including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the pre and post-program assessments at: https://checkrare.com/learning/p-new-guidance-to-diagnose-and-treat-tumor-induced-osteomalacia-tio-2023-1/. Your certificate will be emailed to you in within 30 days. Privacy For more information about the American Academy of CME privacy policy, please access http://www.academycme.org/privacy.htm For more information about CheckRare's privacy policy, please access https://checkrare.com/privacy/ For any questions, please contact: CEServices@academycme.org Copyright © 2023. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare CE grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).

Jointly Provided by American Academy of CME and CheckRare CE Support for this accredited continuing education activity has been made possible through educational grant from Kyowa Kirin. Estimated time to complete: 0.25 hours Start date: November 30, 2023 End date: November 30, 2024 This 15-minute CME-accredited program, hosted by Aliya Khan, MD, Clinical Professor of Medicine at McMaster University, highlights the best practices to diagnose tumor induced osteomalacia (TIO) based on the recently published guidelines in the Journal of Internal Medicine. To earn credit, go to https://checkrare.com/learning/p-new-guidance-to-diagnose-tumor-induced-osteomalacia-tio-2023-2/Activity Faculty Aliya Khan MD, FRCPC, FACP, FACE, FASBMR Clinical Professor of Medicine Director, Calcium Disorders Clinic Director, Fellowship in Metabolic Bone Disease McMaster University Target Audience This activity has been designed to meet the educational needs of physicians specializing in neurology, orthopedics, internal medicine/general practice, rheumatology, endocrinology, pain management, and radiology, who may be involved in the care for individuals with TIO. Other healthcare providers may also participate. Learning Objectives After participating in the activity, learners should be better able to • Describe the latest recommendations for diagnosing patients with TIOAccreditation and Credit Designation In support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is Jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team. Physicians American Academy of CME, Inc., designates this enduring material for a maximum of 0.25 AMA PRA Category 1 CreditsTM. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Other HCPs Other members of the care team will receive a certificate of participation. Disclosure Statement According to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated. Disclosure of relevant financial relationships are as follows: Faculty Educator Dr. Khan discloses the following relevant financial relationships with ineligible companies to disclose: • Advisory Board/Consultant: Amgen, Ascendis, Alexion • Grant/Research support: Ascendis, Alexion, Amolyt • Speakers Bureaus: Amgen, Ascendis, Alexion Planners for this activity have no relevant financial relationships with any ineligible companies. This activity will review off-label or investigational information. The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information. Method of Participation There are no fees to participate in the activity. Participants must review the activity information including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the pre and post-program assessments at: https://checkrare.com/learning/p-new-guidance-to-diagnose-tumor-induced-osteomalacia-tio-2023-2/Your certificate will be emailed to you in within 30 days. Privacy For more information about the American Academy of CME privacy policy, please access http://www.academycme.org/privacy.htm For more information about CheckRare's privacy policy, please access https://checkrare.com/privacy/ Contact For any questions, please contact: CEServices@academycme.org Copyright © 2023. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare CE grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).

Jointly Provided by American Academy of CME and CheckRare CE Support for this accredited continuing education activity has been made possible through an educational grant from Kyowa Kirin. Estimated time to complete: 0.25 hours Start date: November 30, 2023 End date: November 30, 2024 This 15-minute CME-accredited program, hosted by Aliya Khan, MD, Clinical Professor of Medicine at McMaster University, highlights the best practices to diagnose tumor induced osteomalacia (TIO) based on the recently published guidelines in the Journal of Internal Medicine. To earn credit, go to https://checkrare.com/learning/p-new-guidance-to-treat-tumor-induced-osteomalacia-tio-2023-3/ Activity Faculty Aliya Khan MD, FRCPC, FACP, FACE, FASBMR Clinical Professor of Medicine Director, Calcium Disorders Clinic Director, Fellowship in Metabolic Bone Disease McMaster University Target Audience This activity has been designed to meet the educational needs of physicians specializing in neurology, orthopedics, internal medicine/general practice, rheumatology, endocrinology, pain management, and radiology, who may be involved in the care for individuals with TIO. Other healthcare providers may also participate. Learning Objectives After participating in the activity, learners should be better able to • Describe the latest recommendations for treating patients with TIOAccreditation and Credit Designation In support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is Jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team. Physicians American Academy of CME, Inc., designates this enduring material for a maximum of 0.25 AMA PRA Category 1 CreditsTM. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Other HCPs Other members of the care team will receive a certificate of participation. Disclosure Statement According to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated. Disclosure of relevant financial relationships are as follows: Faculty Educator Dr. Khan discloses the following relevant financial relationships with ineligible companies to disclose: • Advisory Board/Consultant: Amgen, Ascendis, Alexion • Grant/Research support: Ascendis, Alexion, Amolyt • Speakers Bureaus: Amgen, Ascendis, Alexion Planners for this activity have no relevant financial relationships with any ineligible companies. This activity will review off-label or investigational information. The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information. Method of Participation There are no fees to participate in the activity. Participants must review the activity information including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the pre and post-program assessments at: https://checkrare.com/learning/p-new-guidance-to-treat-tumor-induced-osteomalacia-tio-2023-3/Your certificate will be emailed to you in within 30 days. Privacy For more information about the American Academy of CME privacy policy, please access http://www.academycme.org/privacy.htm For more information about CheckRare's privacy policy, please access https://checkrare.com/privacy/ Contact For any questions, please contact: CEServices@academycme.org Copyright © 2023. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare CE grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. In the fourth quarter of this year, the FDA is expected to make several important decisions. One of these decisions involves the potential approval of the first CRISPR medicine, which could have significant implications for gene therapy. Additionally, the FDA is considering clearances for companies such as Alnylam, Amgen, and Pfizer, which could lead to the approval of important new therapies.In a major acquisition move, Eli Lilly is set to enter the radiopharmaceutical drug field with a $1.4 billion acquisition of Point Biopharma. This deal will give Lilly access to a pipeline of experimental radioligand therapies and a valuable manufacturing plant. Speaking of leadership changes, Lilly has also announced that Mike Mason will be retiring as the head of its diabetes and obesity division, with Patrik Jonsson taking over.SAS, an analytics company, is encouraging the adoption of open-source technology in the life sciences industry. They emphasize the importance of maintaining security and compliance while utilizing open-source tools.AbbVie has acquired Mitokinin, a Parkinson's drug developer. This acquisition allows AbbVie to expand its portfolio in neurology.Moving on to other news, Amgen has completed its $28 billion acquisition of Horizon after facing challenges from the Federal Trade Commission. However, an FDA panel has found that the data for Amgen's lung cancer drug Lumakras is unreliable, which is a setback for the company. The National Institutes of Health (NIH) has funded research for three experimental drugs for amyotrophic lateral sclerosis (ALS) as part of the ACT for ALS program. Dana-Farber spinout Precede has emerged from stealth with $57 million in funding for its blood test technology.Aetna, the insurance company, may have received overpayments of $25.5 million in Medicare Advantage (MA) for 2015 and 2016. Novant Health has cut 160 jobs due to a challenging healthcare environment. A study published in JAMA Network Open found that physician burnout rates doubled between 2019 and 2021.GE Healthcare Imaging CEO, Jan Makela, discussed the collaboration between GE and Mayo Clinic to advance healthcare through technological innovation. The FDA has called for further tests on recalled devices from Philips due to inadequate testing. A retrospective study by Johnson & Johnson found that catheter ablation is more effective than antiarrhythmic drugs in reducing the risk of heart failure. Northwestern University researchers have found that generative AI models can match the accuracy and quality of radiologist reports.Amgen has successfully completed its acquisition of Horizon and plans to provide updated guidance during its third-quarter earnings call. Mirati Therapeutics is reportedly in acquisition discussions with Sanofi. The Oncologic Drugs Advisory Committee has voted against Amgen's request for full approval of its G12C KRAS inhibitor Lumakras. A study published in JAMA has found that GLP-1 agonists are associated with an increased risk of gastrointestinal adverse events when used for weight loss.In the gene therapy field, Kyowa Kirin is set to acquire Orchard, while uniQure has laid off staff and cut back on research to lower costs. Regeneron and Intellia have expanded their gene editing partnership to target neurological diseases. These developments highlight the ongoing advancements in biopharma and the potential for significant breakthroughs in medicine. Stay tuned for more updates on acquisitions, FDA decisions, cost-cutting measures, and partnerships in the gene therapy field.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.In our first news, we have a summary of an on-demand webinar that discusses how real-world data (RWD) can be leveraged in rare disease research. The use of RWD in clinical trials and drug development for rare diseases is explored by experts from Pfizer and Clarivate. They highlight the importance of RWD in narrowing the gap of knowledge surrounding rare diseases and how it can enhance clinical trials and improve drug development processes. Challenges associated with harnessing RWD effectively are also addressed, along with best practices for overcoming these obstacles. The goal is to optimize rare disease research by leveraging RWD and improving drug development efficiency.Moving on to our next news, we have a promotion for an on-demand webinar titled "High-Throughput Process & Analytical Platforms to Accelerate Gene Therapy Scale-Up." This webinar discusses various topics related to gene therapy scale-up and how innovative platforms can revolutionize the process. Some key points covered include serotype agnostic analytics for AAV manufacturing, challenges in scaling up to larger bioreactors, using an advanced platform with a reduced number of plasmids, and Resilience and Lacerta's innovations. Experts in the field share their insights, and interested individuals can connect with the Resilience team for support in gene therapy process and analytical development work.In regulatory news, the FDA has placed a partial clinical hold on two studies of Innate Pharma's investigational therapy lacutamab following a patient death from the rare blood disease hemophagocytic lymphohistiocytosis. Innate Pharma is working closely with the FDA to address the agency's concerns and provide the necessary data. In other news, an FDA advisory committee has backed the use of US WorldMeds' investigational drug for high-risk neuroblastoma, despite the lack of a randomized controlled trial. This decision could set a precedent for future drug approvals without randomized controlled trial data. Japan-based pharma company Kyowa Kirin is also acquiring Orchard Therapeutics pending regulatory approvals, and Johnson & Johnson's spravato has shown promising results in a phase III head-to-head study.Shifting gears to marketing and advertising, we discuss the importance of connected TV for marketers and how the Hollywood shutdown could disrupt the ad market. Disney's innovative holiday marketing strategies are highlighted, along with Olaplex's unique approach to addressing dupe culture on TikTok. Three Olives Vodka is mentioned for using AI to plan the perfect party, and Meta (formerly known as Facebook) is expanding access to its generative AI tools. Target has launched an immersive toy experience for online shoppers, allowing them to explore a virtual room of products.In the rare disease space, we delve into regulatory challenges related to the use of biomarkers to measure efficacy. The FDA recently approved Novo Nordisk's drug rivfloza for patients with primary hyperoxaluria, but there are concerns about shortcomings in regulatory policies when it comes to biomarkers for rare diseases. Leaders in the pharmaceutical industry who are skilled at translating scientific research into products that regulators see value in are recognized on this year's Pharmavoice 100 list.Lastly, we address the challenges faced by health systems in their workforce due to a volatile economy. Burnout and high turnover rates persist despite efforts to raise wages and strengthen hiring. The nursing shortage is a major issue, and efforts are being made to address the gender pay gap among physicians. On a positive note, there has been a record number of residency matches for 2023, providing insights into current labor trends in healthcare.That wraps up today's episode of Pharma and Biotech daily. Stay tuned for more important news and updates in

Ginette Okoye, MD, FAAD interviewed by Brad Glick, DO, MPH, FAAD

In this podcast episode, Steven M. Horwitz, MD; Pamela B. Allen, MD, MSc; and Kerry Savage, MD, MSc, answer questions from an audience of healthcare professionals on topics related to the management of T-cell lymphomas, including:Use of brentuximab vedotin in ≤1% CD-30‒expressing lymphomasStrategies with JAK inhibition and epigenetic agentsAllogeneic transplantation in cutaneous T-cell lymphomaPresenters: Steven M. Horwitz MDMember, Memorial Sloan Kettering Cancer Center Attending Physician, Memorial HospitalProfessor of Medicine, Weill Cornell Medical College New York, New YorkPamela B. Allen, MD, MScAssistant ProfessorDepartment of Hematology/OncologyEmory University, Winship Cancer InstituteThe Emory ClinicAtlanta, GeorgiaKerry Savage MD, MScProfessor of Medicine University of British ColumbiaMedical OncologistDivision of Medical OncologyBC Cancer Vancouver, British Columbia, CanadaContent for this program was supported by educational grants from Daiichi Sankyo; Kyowa Kirin, Inc.; and Seagen Inc. Link to the full program, including downloadable slides, expert commentary, and an on-demand webcast:bit.ly/3ItqEOd

Synopsis: This special episode features a panel discussion with three biotech leaders about drug development for rare diseases. James Mackay, Ph.D., is the Founder, President and CEO of Aristea Therapeutics, an immunology-focused clinical stage biotech that is focused on inflammatory diseases. Arthur T. Suckow, Ph.D., is the Co-Founder and CEO of DTx Pharma, which is focused on developing novel technology for delivery of RNA medicines. Eslie Dennis is the SVP and CMO of Kyowa Kirin North America, a Japan-based global specialty pharmaceutical company. Our guests discuss the patient journey for those living with rare diseases, the importance of driving awareness for this patient population, important points to consider when partnering with big pharma, clinical trial design for patients with rare diseases, and best practices for driving awareness and inclusion of people of color in rare disease trials. Biography: James Mackay, Ph.D., Founder, President and Chief Executive Officer, has over 25 years of development and commercialization expertise in the pharmaceutical industry, including 6 drug product approvals across multiple therapeutic areas. Prior to founding Aristea, he was President and Chief Executive Officer of Ardea Biosciences, Inc., following the company's acquisition by AstraZeneca in 2012. James was instrumental to setting up an innovative model for Ardea Biosciences that retained the biotech's independence and accountability for the development of the gout franchise while also developing a synergistic and collaborative relationship with the parent company, AstraZeneca. Prior to Ardea, James held senior executive positions at AstraZeneca where he led the development and commercialization of drugs across a range of therapy areas. and managed significant global functional departments. James plays a pivotal role in the San Diego Ecosystem and currently sits on the Board and Executive Committee of CONNECT and sits on the Board of BIOCOM. He is a former Board member of the San Diego Economic Development Corporation (EDC). James holds a BS in Genetics and Ph.D. in Medical Genetics from Aberdeen University, Scotland. Dr. Arthur Suckow founded DTx Pharma in 2017. An innovative leader in drug discovery, he previously worked on the ophthalmology, diabetes, and NASH programs at Regulus Therapeutics, at the MedImmune arm of AstraZeneca, and in the Diabetes Drug Discovery program at Johnson & Johnson. Dr. Suckow received his BS from the University of Delaware and his PhD from University of California, San Diego. He has received numerous awards including a Beckman Fellowship, a NSF graduate research fellowship, and a BIOCOM catalyst award. Dr. Eslie Dennis joined Kyowa Kirin in May 2021 as SVP, Chief Medical Officer for Kyowa Kirin North America. Prior to joining this organization, she was Vice President and Head Global Medical Affairs at Ventana/Roche Tissue Diagnostics. She is a physician with 10 years experience in clinical practice (internal medicine and hematology/oncology) and research, and over 20 years international biopharma experience holding positions of increasing responsibility in pharmaceutical and diagnostic organizations at MSD, Novartis and Roche, including leadership of public-private consortia at the Critical Path Institute. Throughout her career, Dr. Dennis has championed innovative science and solutions to address unmet needs for patients and society, particularly addressing healthcare disparities. Dr. Dennis received her MBChB from the Godfrey Huggins School of Medicine in Harare, Zimbabwe, and was the recipient of the Winston Churchill, Margaret Low, and Prankard-Jones Scholarships, as well as the Guy Elliot Bursary. She is a Fellow of the College of Physicians of South Africa and received her internal medicine and hematology/oncology training at Groote Schuur Hospital in Cape Town, South Africa.

A few months ago I spoke live across social media with four colleagues, experts and Medical Affairs senior leaders with diverse, global perspectives across pharma and medical devices to discuss some of the best moments and surprises from the Medical Affairs Professional Society's (MAPS) May 2022 Meeting in Paris which we attended. The conversation was a recap of this meeting yet with lots of very forward looking concepts many of you, my friends and colleagues, may enjoy reviewing, particularly as we prepare for the 2023 European meeting to be held in Lisbon by MAPS. So listen to this conversation with Marc Sirockman, Global Chief Executive Officer of MedEvoke; Meg Heim, President and Founder of Heim Global Consulting; Marcus West CEO and Co-Founder of 60seconds.com, and Viorica Braniste, Medical Excellence and Patient Engagement Director at Kyowa Kirin, which emphasizes some of the most pressing issues our Medical division is facing in the industry as we anticipate more developments in our Age of Information. Meg Heim highlights innovative qualitative research in R & D demonstrating Medical Affairs' value earlier in a product lifecycle from Kyowa Kirin as an example of how far along the drug development pipeline the division is able to impact with meaning. Marc reminds the group about the powerful turning point Medical Affairs is undergoing presently, summarized by the meeting's theme "The Medical Affairs' Time to Lead is Now". He mentions that while digital information has exploded over the last two years, Medical has outgrown other functions and it is critical to communicate its value internally to receive the appropriate funding and resources. Marcus speaks about the need for enhancing the coaching culture and real time feedback in Medical - a recurring theme at the meeting. Viorica addresses the unmet need of coaching and training to connect medical insights with powerful business impact to critical diverse stakeholders. Throughout our discussion, we concede how fragmented Medical continues to be in its organization, and why we must change our communication to be more consistent with our environment. We recall an inspirational quote from the meeting shared by Ben Bryant, a veteran pharma executive coach, who said "Leaders are not paid to make the inevitable happen. They are paid to make happen what would otherwise not happen". Our group refers to several sessions about shortening the lag time from generating to capturing to communicating insights, and we compare raw medical insights to oil, which if "unrefined" by definitions or context, become difficult to translate into value. Thus, Marc encourages pharma to begin to "ask for help" more because data insights are becoming overwhelming to manage alone without various agencies and life science solution providers. We also discuss intriguing, controversial, but also encouraging moments from the meeting such as the growing critical need of "ROI" of Medical and performance metrics as well as new conversations about Medical's potential in guiding an entire organization in improving the accuracy of their Sales forecasts. Avishek Pal, Scientific Communications Director at Novartis Oncology in Basel, Switzerland, relays his impressions of the importance of the "cross functional co-creation journey" between Medical Education, Field Medical as well as their insights, and multi-channel and formats of Medical Education to improve how pharma reaches healthcare professionals across the globe. So we look forward to more conversations on leadership, metrics, mentorship, as well as prescriber and patient engagement and education perspectives (comment on our discussion from Gorana Dasic, a pharma executive from Pfizer), at the upcoming 2023 meeting in Lisbon as well. Finally, we discuss the value of joining the incredible non-profit MAPS society, its Mentorship program, and vote on our favorite cities for future meetings after Lisbon: Florence, Berlin, Milan, Rome, Tbilisi, Dubrovnik. --- Support this podcast: https://anchor.fm/alloutcoach/support

Crohn's approval, new VC round, direct offerings, and potential asset sales. Find out more athttps://LifeScienceTodayPodcast.comStory ReferencesAbbVieThird RockScholar RockKyowa Kirin About the ShowLife Science Today is your source for stories, insights, and trends across the life science industry. Expect weekly highlights about new technologies, pharmaceutical mergers and acquisitions, news about the moves of venture capital and private equity, and how the stock market responds to biotech IPOs. Life Science Today also explores trends around clinical research, including the evolving patterns that determine how drugs and therapies are developed and approved. It's news, with a dash of perspective, focused on the life science industry. 

Vascular Ehlers Danlos Syndrome (Vascular EDS/VEDS) is just one of 13 sub-types of a group of connective tissue disorders called Ehlers Danlos. Due to a deficiency in collagen the walls of blood vessels are prone to dissection, rupture or aneurysm with potentially fatal consequences. However considering the serious complications of vascular EDS its presentation can be subtle and hard to spot. Clare, Jared and Dr Paddy Coughlin explain how patients with vascular EDS may present and what challenges they face. Trigger warning - descriptions of vascular events and bleeding.And here endeth Season 2! Join us in September for the next Season! LinksAccompanying blog with images about vascular EDS. Annabelle's Challenge aims to promote awareness and medical research into the rare, life-threatening and incurable genetic condition Vascular Ehlers-Danlos Syndrome (Vascular EDS). It was founded in 2013 by Jared and Sarah Griffin, after their daughter Annabelle was diagnosed with Vascular EDS at the age of 3.Please watch this short video where you can see Jared and Sarah talk about Annabelle's diagnosis. As well as hear other families share their stories.Ehlers Danlos Support UK (EDS UK) was set up in 1987 to support, advise and inform those living with the Ehlers-Danlos syndromes.  The charity represents and supports people with all types of EDS.Ehlers Danlos GP Toolkit Orphanet's description of Vascular Ehlers DanlosLoeys-Dietz is another connective tissue disorder that Paddy mentioned. Loyes-Dietz also causes aneurysms as well as other features. Von Willebrand Disorder (VWD) is a common misdiagnosis given to people with vascular EDS. VWD is a clotting disorder unlike vascular EDS which is a connective tissue disorder.  People with VWD have low levels of a protein involved in blood clotting, von Willebrand Factor (VWF) in their blood, or their VWF is dysfunctional, so it takes longer for the clotting process to work and for bleeding to stop.Sponsorship NoticeThis podcast is brought to you by Medics4RareDiseases. M4RD receives sponsorship from commercial companies. In 2022 this includes:Alexion Therapeutics, Amicus Therapeutics UK Ltd, BioMarin, Bionical Emas, Healx, Kyowa Kirin, Orchard Therapeutics and SOBI.These companies have no editorial control over this or any other M4RD content. Sponsorship does not equate to endorsement of companies or products. Views expressed during this podcast are personal and don't reflect those of M4RD sponsors.Go here to find out more about how M4RD works with sponsors. Companies have no editorial control over any of M4RD's activities or content of this podcast.

Lucy and Melissa record live from emotive agency offices in London! Joined by two special guests, Jason and Kelly from the Chinese University of Hong Kong, who have taken a break in the medical studies to see how rare disease education and advocacy is approached in other countries.Part One: Melissa Clasen, Education and Training Officer for M4RD, talks about what a genetic counsellor does as well as who can access this service and how to make that happen. Melissa trained in genetic counselling in South Africa and moved to the UK 4 years ago. Before joining M4RD, Melissa worked at Great Ormond Street Hospital in London. Part Two: Jason and Kelly introduce themselves, what they've been learning, what medical school is like in Hong Kong.LinksNHS information on genetic counsellingBritish Society of Genetic MedicineGenomics Education ProgrammeRare Disease Hong KongUnique - information for families and professionals about rare chromosomal conditionsSponsorship NoticeThis podcast is brought to you by Medics4RareDiseases. M4RD receives sponsorship from commercial companies. In 2022 this includes:Alexion Therapeutics, Amicus Therapeutics UK Ltd, BioMarin, Bionical Emas, Healx, Kyowa Kirin, Orchard Therapeutics and SOBI.These companies have no editorial control over this or any other M4RD content. Sponsorship does not equate to endorsement of companies or products. Views expressed during this podcast are personal and don't reflect those of M4RD sponsors.Novartis support M4RD's Mystery Monday.Go here to find out more about how M4RD works with sponsors. Companies have no editorial control over any of M4RD's activities or content of this podcast.

Melissa from M4RD interviews Marie who was a healthy and active paramedic until one shift when she suddenly started experiencing pain in her left side (left upper quadrant pain). As a healthcare professional Marie knew likely causes for the sudden pain and knew how to treat it. However the wasn't musculoskeletal or costochondritis or even cholesystitis. It was in fact caused by compression of the duodenum by two arteries. A condition known as Superior Mesenteric Artery Syndrome (SMA - but not spinal muscular atrophy!!)Marie talks about the pursuit of a diagnosis. What it did and didn't give her. How her mental health has been negatively impacted by the "rollercoaster" she has been on and how her family have been affected.As an HCP herself, Marie is pragmatic about the challenges faced by her peers in the NHS and reflects on what she has learned from her own experiences. Which she uses in her role as Teaching Fellow in paramedic Practice at Oxford Brookes University. Find out more about the course here.LinksRead our latest blog A REAL rollercoaster - from paramedic to patientMore info on Superior Mesenteric Artery Syndrome from NORDRenal Nutcracker Syndrome on Orphanet - a related compression syndrome of the left renal vein (LRV) between the superior mesenteric artery (SMA) and the abdominal aortaNicolette Baker suffered from SMA Syndrome for years causing her nausea and she was unable to eat. She went undiagnosed for years and was going for surgical treatment in Germany before she sadly died.Sponsorship NoticeThis podcast is brought to you by Medics4RareDiseases. M4RD receives sponsorship from commercial companies. In 2022 this includes:Alexion Therapeutics, Amicus Therapeutics UK Ltd, BioMarin, Bionical Emas, Healx, Kyowa Kirin, Orchard Therapeutics and SOBI.These companies have no editorial control over this or any other M4RD content. Sponsorship does not equate to endorsement of companies or products. Views expressed during this podcast are personal and don't reflect those of M4RD sponsors.Go here to find out more about how M4RD works with sponsors. Companies have no editorial control over any of M4RD's activities or content of this podcast.

This is Part 2 of a live recording of The Unusual Suspects: Rare disease in everyday medicine which was hosted by M4RD and The Medical Genetics Section of The Royal Society of Medicine in February 2022. The Royal Society of Medicine event page for The Unusual Suspects 2022In this second part you will hear about:The role of genomics in achieving health equityDr Denise Williams, Consultant Clinical Geneticist, Birmingham Women's and Children's NHS Foundation TrustEnhanced Genetics Services Evaluation ReportGenome UK: The future of healthcareReading recommendation!! The Immortal Life of Henrietta LacksNarrative based medicine and intersectionalityDr Sondra Butterworth, Community Psychologist, RareQoLListen to Sondra talking about inclusivity in Season 1 of the podcast hereThe Whose Voice is it Anyway report that was produced by RareQoL , M4RD & NHS England.Improving knowledge and understanding of rare disease: Lessons learned from an an unusual heart attackDr David Adlam, Associate Professor of Acute and Interventional Cardiology, University of LeicesterListen to the episode featuring Dave and BeatSCAD called Not your usual heart attack herePutting the ‘I' in Intersectionality: the unspoken pandemicMiss Zainab Alani, Medical Student, University of Glasgow & The Student Voice Prize 2021 winner Learn more about Myasthenia Gravis through myaware and OrphanetSponsorship NoticeM4RD was able to produce this event thanks to sponsorship by by Alexion Therapeutics and SOBI. Neither company has any editorial control over content and sponsorship does not equate to endorsement. Views expressed in this episode are personal and do not reflect those of the companies involved.This podcast is brought to you by Medics4RareDiseases. M4RD receives sponsorship from commercial companies. In 2022 this includes:Alexion Therapeutics, Amicus Therapeutics UK Ltd, BioMarin, Bionical Emas, Healx, Kyowa Kirin, Orchard Therapeutics and SOBI.These companies have no editorial control over this or any other M4RD content. Sponsorship does not equate to endorsement of companies or products. Views expressed during this podcast are personal and don't reflect those of M4RD sponsors.Go here to find out more about how M4RD works with sponsors. Companies have no editorial control over any of M4RD's activities or content of this podcast.

This is Part 1 of a live recording of The Unusual Suspects: Rare disease in everyday medicine which was hosted by M4RD and The Medical Genetics Section of The Royal Society of Medicine in February 2022.In this first part you will hear from Dr Lucy McKay on Rare Disease 101 and Aisha Seedat on The UK Rare Disease's Framework. Aisha lives with mucopolysaccharidosis type IV (Morquio Syndrome) and she reflects on what she would like to see from the Framework. M4RD was able to produce this event thanks to sponsorship by by Alexion Therapeutics and SOBI. Neither company has any editorial control over content and sponsorship does not equate to endorsement. Views expressed in this episode are personal and do not reflect those of the companies involved. LinksThe Royal Society of Medicine event page for The Unusual Suspects 2022The UK MPS SocietyThe Whose Voice is it Anyway report that Aisha contributed to.The Sickle Cell Society and the APPG's No-One's Listening Report  published following inquiry into avoidable deaths and failures of care for sickle cell patients.DeclarationDr Lucy McKay is on the Faculty Steering Committee for Medscape's Pathways for Rare Disease. She has received an honorarium in order to guide and develop this resource.Sponsorship NoticeThis podcast is brought to you by Medics4RareDiseases. M4RD receives sponsorship from commercial companies. In 2022 this includes:Alexion Therapeutics, Amicus Therapeutics UK Ltd, BioMarin, Bionical Emas, Healx, Kyowa Kirin, Orchard Therapeutics and SOBI.These companies have no editorial control over this or any other M4RD content. Sponsorship does not equate to endorsement of companies or products. Views expressed during this podcast are personal and don't reflect those of M4RD sponsors.Go here to find out more about how M4RD works with sponsors. Companies have no editorial control over any of M4RD's activities or content of this podcast.

Our episode features two leaders at the global specialty pharmaceutical company Kyowa Kirin. Kyowa Kirin has a particular focus on the discovery and development of novel, first-in-class medicines that have a profound impact on patients in multiple therapeutic areas. Rachel Soloff is an executive director of research with expertise in immunology and the discovery of novel monoclonal antibodies and small molecule therapeutic compounds for autoimmune and inflammatory diseases. She oversees the KK US-Research activities, which are focused on target discovery and validation, and leads candidate discovery for multiple innovative pipeline projects. Andrew McKnight currently holds the position of president and CSO at Kyowa Kirin Pharmaceutical Research. He is responsible for early-stage drug discovery within Kyowa Kirin's Immunology & Allergy division. In this episode, we discuss the various clinical and commercial products discovered and developed at Kyowa Kirin. Through Innovative approaches to antibody engineering, KK has been able to consistently maintain a pipeline of products to treat a wide variety of disorders.Hosted by Joe Varriale and Gustavo Carrizo.

Aged six Alexander developed a squint and hearing loss which didn't cause too much alarm until he started displaying strange behaviours akin to dementia such forgetting where his bedroom was.  Despite concerns from Alex's parents, school and grandparents these symptoms were initially dismissed by the local GP. Sadly Alexander was not seen urgently for what would turn out to be a progressive condition that could only be mitigated by timely treatment with a bone marrow transplant.Aged six both Alex and his twin brother, Cameron, were diagnosed with the devastating condition, cerebral leukodystrophy. This is an x-linked condition that they had inherited through their mum, Karen, who was 40 weeks pregnant with her third son at the time.The following events are unimaginable for anyone who's not lived through it and, Karen says, looking back she also finds it hard to believe what happened. Karen and her husband were given Alexander's diagnosis on a Friday afternoon and received no support or follow up over the longest weekend of their lives. But their GP stepped up and literally turned up at their house to say he and his colleagues would be there for whatever they needed.This one small act set the tone for a supportive relationship from then on, putting the diagnostic odyssey behind them, and looking to the future as a partnership in the family's holistic care. This plus peer support through Alex The Leukodystrophy Charity were lifelines and continue to be lifelines for Karen and her family.Links  The Alex Leukodystrophy Charity (Alex TLC)Improving clinical care by learning about lived experience - a talk by Karen Harrison and Dr Alexandra Downes.Sponsorship NoticeThis podcast is brought to you by Medics4RareDiseases. M4RD receives sponsorship from commercial companies. In 2022 this includes:Alexion Therapeutics, Amicus Therapeutics UK Ltd, BioMarin, Bionical Emas, Healx, Kyowa Kirin, Novartis, Orchard Therapeutics and SOBI.These companies have no editorial control over this or any other M4RD content. Sponsorship does not equate to endorsement of companies or products. Views expressed during this podcast are personal and don't reflect those of M4RD sponsors.Go here to find out more about how M4RD works with sponsors. Companies have no editorial control over any of M4RD's activities or content of this podcast.

Lucy is joined by Helena Baker who was born with a congenital limb defect, worked as a nurse and is the outgoing CEO of Rare Disease Nurse Network.Helena was born with fibular hemimelia - a disorder of limb budding results in a congenital limb malformation characterized by complete or partial absence of the fibula bone combined with dysplasia and hypoplasia of the tibia and dysplasia, hypoplasia or aplasia of parts of the foot (Orphanet).Despite having 100s of operations and having a fairly classical presentation of this condition Helena did not know her diagnosis until aged 49.5 years. Despite the name of the condition not changing her management it meant a lot to her family to be able to understand that nobody was to blame for what Helena had endured her whole life. And not long after Helena had to come to terms with the requirement to amputate her "little foot".In this episode Lucy and Helena talk openly about how an assumption of knowledge left her in the dark about her diagnosis, what it's like to say goodbye to your own limb and how coordination of care and better communication could make all the difference to people like Helena. Which is why she is passionate that RDNN become the Macmillan equivalent for rare disease. Sponsorship NoticeThis podcast is brought to you by Medics4RareDiseases. M4RD receives sponsorship from commercial companies. In 2022 this includes: Alexion Therapeutics, Amicus Therapeutics UK Ltd, BioMarin, Bionical Emas, Healx, Kyowa Kirin and SOBI. These companies have no editorial control over this or any other M4RD content. Sponsorship does not equate to endorsement of companies or products. Views expressed during this podcast are personal and don't reflect those of M4RD sponsors. Go here to find out more about how M4RD works with sponsors. Companies have no editorial control over any of M4RD's activities or content of this podcast.

Sian is an inspiring, authentic, and "human-centered" HR professional & expert. Currently, Sian works at Kyowa Kirin, a Japanese pharma group that develops innovative specialty drugs to raise the health and well-being of people around the world. In this episode, you will find out: What strictly a human-centred business looks like The initiatives that Kyowa Kirin have put in place to look after its people How HR have strategized the suitable approaches to take How to get buy-in from stakeholders around putting in more schemes which look after its people And lots more! Contact Sian here - Sian Abel | LinkedIn

Do you know what DIDMOAD stands for? Get your medical dictionary ready...diabetes insipidus, diabetes mellitus, optic atrophy and deafness aka Wolfram Syndrome. This week Lucy is joined by Abby who is a young person living with Wolfram Syndrome, Tracy whose daughter has WS leading her to found Wolfram Syndrome UK with her husband and finally prof Tim Barrett from Birmingham Women's and Children's Hospital who is a Children's Diabetes and Endocrine Consultant.We explore the issues of diagnosis, variability in Wolfram Syndrome, living with complex needs and an invisible condition as well as research in rare disease. We really packed it into this episode!Wolfram Syndrome UKDiabetes GenesOrphanetGene ReviewsClinical trials.govThis activity is partially funded by:Alexion Therapeutics, Amicus Therapeutics UK Ltd, BioMarin, Bionical Emas, Healx, Kyowa Kirin and SOBI.  Sponsorship does not equate to endorsement of any company or its products Go here to find out more about how M4RD works with sponsors. Companies have no editorial control over any of M4RD's activities or content of this podcast.

Lucy welcomes you back to The Rare Disease Podcast 4 Medics for season 2! Yay! This episode explains what M4RD has been up to this year so far and some plans for the future.The Unusual Suspects: Rare disease in everyday medicine was on 9th Feb. Soon you will be able to catch up on all the talks via the M4RD Video Library. Make sure you're on our our mailing list so you don't miss out.The winner of The Student Voice Prize 2021 was announced in February. Congratulations to the winner and runners up. Check out their essays here and if you didn't take part in this essay competition in 2021, 2022 can be your year. Check it out in October.The Guardian Rare Disease Day pullout. Find M4RD and Medscape on page 8 'Rare disease education is key to enabling medics to achieve the best for their patients'. You can check out Medscape's Pathways in Rare Disease here.Co-ordinated care for people affected by rare diseases: the CONCORD mixed-methods study published today!And Lucy thanks M4RD's sponsors of their 2022 activities:Alexion Therapeutics, Amicus Therapeutics UK Ltd, BioMarin, Bionical Emas, Healx, Kyowa Kirin and SOBI.  Sponsorship does not equate to endorsement of any company or its products Go here to find out more about how M4RD works with sponsors. Companies have not editorial control over any activities or content of this podcast.

Zack is currently serving as a hematology/oncology medical science liaison (MSL) with Kyowa Kirin (primary focus in hematologic malignancies). His previous experience included supporting GSK Oncology in a similar liaison capacity. Prior to these field medical roles, Zack had a brief stint in medical writing with Vaniam Group (oncology-focused scientific communications). Zack's first role after pharmacy school came as a clinical content position where he supported and helped to manage the College of American Pathologists (CAP) Cancer Protocols. He was able to work alongside pathologists and key opinion leaders (KOLs) who were cancer subspecialty experts and the authors of these protocols. The Cancer Protocols are used by pathologists worldwide as guidelines for collecting and reporting information on malignant tumor classification, histology, grading, staging, margins, nodal involvement, metastatic disease, biomarker profiles, etc. The Cancer Protocols serve as the gold standard in reporting for not only accreditation purposes but are also instrumental in driving the direction of care as a patient begins the journey from a cancer diagnosis to treatment and beyond.

Complex manufacturing processes and supply chains have always been part of the biopharma industry story, with many people in more than a few nations typically involved along the way. While that observation applies to plenty of other areas of business, Paul Testa, executive vice president for operations and supply chain at Tokyo-based Kyowa Kirin, tells us about the crucial difference with medicine: “We can wait for toilet paper or building materials, but the patient cannot wait because their health care is at stake,” he said. Continuity, reliability of supply, business process and automation were all key priorities for him before the pandemic and haven't drastically changed since. But “there's an added layer of complexity with COVID and a lot of the intricacies of managing those details that we all deal with,” he said. How does the Japanese concept of 和 (wa) fit in? Listen to find out. See acast.com/privacy for privacy and opt-out information.

Lauren Walrath is Vice President of Public Affairs in North America, and Susan Thiele is Director of Advocacy and Brand Communications at Kyowa Kirin, a Japan-based global specialty pharmaceutical company known for its cutting-edge scientific discoveries in four therapeutic areas including neurology, nephrology, hematological cancers, and immunology. Lauren explains their approach to patient-centric discovery, "In advocacy, we're really trying to find where the gaps are and how we can best work with partners to make long-term and sustainable improvements for patients. So that can be in the form of starting new research, funding new grants, addressing disparities, working on developing new education programs. Generally, we're looking for meaningful opportunities to help patients and their families on their journey from diagnosis to treatment. We work closely with the advocates, and we bring all of that understanding back to our teams here at Kyowa Kirin so that we really can engage our cross-functional partners in doing more to help the patients we serve." Susan elaborates, "And then there is Jim and Jeffrey who have Sézary syndrome, which is a more serious form of CTCL that affects less than 5% of the patients. What we've done is we've captured their journeys in their own voice, the journey to diagnosis, their experience on treatment, and ultimately what they've learned along the way, what really keeps them motivated to not give up. And people can now hear those stories by going to POTELIGEO.com." #KyowaKirin #KKNA #DrugDevelopment #RareDiseases #Antibodies #immunology #Cancer #CTCL #Neurology #ParkinsonsDisease #NeurodegenerativeDiseases #Nephrology #HematologicalCancers #SezarySyndrome KyowaKirin.com Download the transcript here

Lauren Walrath is Vice President of Public Affairs in North America, and Susan Thiele is Director of Advocacy and Brand Communications at Kyowa Kirin, a Japan-based global specialty pharmaceutical company known for its cutting-edge scientific discoveries in four therapeutic areas including neurology, nephrology, hematological cancers, and immunology. Lauren explains their approach to patient-centric discovery, "In advocacy, we're really trying to find where the gaps are and how we can best work with partners to make long-term and sustainable improvements for patients. So that can be in the form of starting new research, funding new grants, addressing disparities, working on developing new education programs. Generally, we're looking for meaningful opportunities to help patients and their families on their journey from diagnosis to treatment. We work closely with the advocates, and we bring all of that understanding back to our teams here at Kyowa Kirin so that we really can engage our cross-functional partners in doing more to help the patients we serve." Susan elaborates, "And then there is Jim and Jeffrey who have Sézary syndrome, which is a more serious form of CTCL that affects less than 5% of the patients. What we've done is we've captured their journeys in their own voice, the journey to diagnosis, their experience on treatment, and ultimately what they've learned along the way, what really keeps them motivated to not give up. And people can now hear those stories by going to POTELIGEO.com." #KyowaKirin #KKNA #DrugDevelopment #RareDiseases #Antibodies #immunology #Cancer #CTCL #Neurology #ParkinsonsDisease #NeurodegenerativeDiseases #Nephrology #HematologicalCancers #SezarySyndrome KyowaKirin.com Listen to the podcast here

Welcome to the next episode of The Rise of The Customer Podcast, the go-to podcast for CX professionals looking to transform their organisation. In this episode, we're joined by Danie du Plessis, Executive Vice President of Medical Affairs at Kyowa Kirin International and Board Member of the Medical Affairs Professional Society (MAPS). With 28 years in multinational pharma companies, including a FTSE 100 company, Danie has a successful track record of people development, leadership and managing change so we're delighted to have him on the podcast. During his conversation with Neil Sharp, Danie explores: ➡️ The role of medical affairs in the world of pharma ➡️ How consumerism, access to information and new technologies are propelling change ➡️ The importance of harvesting and understanding data to drive customer experience ➡️ The impact of tech giants such as Google and Amazon on the healthcare sector If you're a pharma leader looking to gain insight from one of the industry's most experienced experts, this episode is not to be missed.

Dr. Maggie Smith works at Kyowa Kirin as a National Director within Medical Affairs, leading the North America Oncology Medical Science Liaison (MSL) program.  Dr. Smith's clinical knowledge comes from her first-hand experience at the prestigious University of Chicago Hospitals, where she worked as a, Charge Nurse on the Bone Marrow Transplant (BMT) overflow unit and as a Clinical Research Associate, working on various Phase 1-3, oncology clinical trials and currently she still practices per diem.   Dr. Smith also writes for an online oncology nursing journal, highlighting oncology topics in the Oncology Nursing News.  --------------------------------------------------------------------1.  Click the link to learn more about the Nurse Wellness Mentorship2. Download your FREE Mindfulness E-Book at stressblueprint.com/353. Follow the Nurse Wellness Podcast on Facebook and Instagram4. Email Nurse Wellness Podcast at hello@stressblueprint.com5. Background music produced by DNMbeats