Podcasts about hemophilia

With Pfizer discontinuing Beqvez and BioMarin scaling back the commercial focus of Roctavian, the curative promise of hemophilia gene therapies is tempered by significant barriers that discourage widespread adoption. In this week’s episode of “The Top Line,” Fierce Pharma’s Zoey Becker speaks with Glenn Pierce, M.D., Ph.D., vice president of medical at the World Federation of Hemophilia, about the complexities behind these innovative therapies and the multifaceted reasons for their slow uptake. Pierce discusses the competitiveness of the current hemophilia gene therapy market, why Pfizer’s product couldn’t keep up, and the patient populations that risk being left behind. To learn more about the topics in this episode: As Pfizer backs out of hemophilia gene therapy space, CSL hopes Hemgenix is here to stay Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio BioMarin downsizes Roctavian efforts but keeps hemophilia gene therapy for 3 markets See omnystudio.com/listener for privacy information.

This episode of the Global Hemophilia Report explores the long-overdue conversation about women with hemophilia. Experts discuss the challenges women face in getting diagnosed, the persistent gaps in clinical research and care, and the impact of outdated language and systemic bias. The panel highlights the need for better data, more inclusive clinical trials, and a shift in medical culture to ensure women with hemophilia are recognized and prioritized.    Guests: Michelle Sholzberg MDCM, FRCPC, MSc. Dawn Rotellini Len Valentino, MD Robert Sidonio, Jr., MD   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Written by: Kay Vermeil   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes:   Presenting Sponsor: Presented by Sanofi Join Shellye as she shares her inspiring journey as a woman living with hemophilia. After years of unanswered questions, her diagnosis highlights the critical role of health literacy in bridging care gaps. Through advocacy, Shellye raises awareness and empowers underserved communities. Watch her story and see how health literacy can break barriers and transform lives. Click here to watch her story: Sanofi – See Hemophilia Through My Eyes: Women & Girls For too long, women and girls who bleed have been dismissed. Left out of the narrative. Ignored by the system. But not anymore. In our new film, “Dismissed,” meet Isabelle—a 15-year-old with hemophilia who's using her voice to uplift the unheard. Alongside her are four powerful stories of women challenging what's "normal" and demanding recognition, care, and justice. This is more than a film. It's a movement.

Joining us on Well Said is Dr. Suchitra Acharya, a Pediatric Hematologist/Oncologist, Director of the Hemostasis and Thrombosis Center at LIJMC, Head of the Bleeding Disorders and Thrombosis Program at Cohen Children's Medical Center, and Professor of Pediatrics at the Zucker School of Medicine at Hofstra/Northwell. Dr. Acharya, along with two of her patients, will […]

In this episode of the Global Hemophilia Report, host Patrick James Lynch and a panel of experts discuss the importance of real world data and patient engagement in hemophilia care. The conversation explores how data collected outside of clinical trials provides deeper insights into treatment outcomes, challenges, and lived experiences. Guests share strategies for improving data reliability, motivating patient participation, and balancing privacy with research needs. Tune in for key takeaways on how both numbers and personal stories shape better care for the hemophilia community.   Guests: Mike Recht, MD, PhD, MBA Samantha Gouw, MD, PhD Maria Santaella, RN-BC, MSN, PhD(c)   Senior Advisor: Donna DiMichele, MD   Hosted by: Patrick James Lynch   Written by: Kay Vermeil   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes:   Presenting Sponsor: Presented by Sanofi   Sanofi's Global Hemophilia Survey uncovers significant care gaps and emotional challenges faced by patients and caregivers. Learn how improving health literacy and fostering better patient-provider communication are essential to addressing these inequities. Explore the findings and see how Sanofi is driving health equity for the hemophilia community. Explore the survey findings here: Global Hemophilia Survey Page. For too long, women and girls who bleed have been dismissed. Left out of the narrative. Ignored by the system. But not anymore. In our new film, “Dismissed,” meet Isabelle—a 15-year-old with hemophilia who's using her voice to uplift the unheard. Alongside her are four powerful stories of women challenging what's "normal" and demanding recognition, care, and justice. This is more than a film. It's a movement.

Dive into hemophilia with three-peat guest Dr. Weyand, the Shematologist! In this episode, we break down the basics of hemophilia A and B, from inheritance and pathophysiology to the latest advancements in diagnosis and treatment. Factor in these practical tips for managing acute bleeding, counseling families, and navigating the complexities of prophylaxis—all while keeping kids safe and active. The pearls just kept flowing and flowing!

BONUS: Patrick James Lynch on Entertainment That Makes Change - Lessons in Product Thinking from Believe Ltd. In this BONUS episode we explore how Patrick James Lynch, filmmaker, media executive, and rare disease advocate, has built Believe Limited around a powerful mission: entertainment that effects change. Patrick shares his journey from personal experience with his brother's hemophilia to creating award-winning content that empowers rare and chronic disease communities, offering valuable lessons for product managers on human-centered design, stakeholder alignment, and building emotionally viable products. The Genesis of Entertainment That Effects Change "This is more than a product." Patrick's journey began with a deeply personal question about his brother who had hemophilia. As an entrepreneur, he set out to respond to an identified need with one product to meet that need, but quickly realized the scope was much larger. His curiosity about what was different between him and his brother led him to understand that he needed to help people like his brother. This realization drove him to create valuable online videos to engage their audience, marking the beginning of Believe Ltd.'s mission of entertainment that effects change. Essential Product Lessons: Listen, Learn, and Do No Harm "The fact that I am my audience, does not mean that I'm an expert." Patrick emphasizes the critical importance of conducting thorough needs assessments and truly understanding your community before building products. Key insights include: Embed yourself in the community you're serving rather than making assumptions Follow the principle of "listen, learn and do no harm" as your starting point Involve community engagement as a dedicated role - Believe Ltd. has a VP of community engagement Define clear phrases that explain the value you deliver to your audience Use your personal story to establish credibility and relate experiences to your audience The goal is to get as familiar with your community as possible, then conduct your own research and development based on those deep insights. Navigating Multi-Stakeholder Complexity "Collaboration only succeeds when all points of view are respected." Working with patients, funders, healthcare professionals, and pharmaceutical companies requires careful orchestration. Patrick's approach centers on prioritizing the end game and identifying the north star goal that aligns all parties. He emphasizes focusing on combined skills and networks rather than trying to accomplish everything at the start. The key is ensuring that aligning stakeholders becomes a central part of the process, with everyone being accounted for throughout the journey. Human-Centered Storytelling as Product Strategy "What's the story that shows the value add of your product?" Patrick advocates for human-centered storytelling as a fundamental product approach. Rather than leading with features or specifications, he suggests crafting stories that demonstrate real value - like how a thermos saved someone's life while hiking. Stories have been humanity's primary communication tool since the beginning of time, and they remain the most effective way to show product value and connect with audiences on a meaningful level. Being a Value Fundamentalist "At any given moment, if anyone takes a screen grab, and set it against our five core values as a company - you see it's playing out." Patrick describes himself as a value fundamentalist, meaning that their company's core values are always present in everything they do. This requires courage, including the willingness to say "no" when opportunities don't align with their values. As CEO, he believes in embodying these values consistently, even when it's challenging, because who they are must always be visible in their work. Balancing Vision with Community Feedback "When you ask the audience for a solution, there's no innovation." Patrick warns against sacrificing vision simply because you're working closely with your audience. While being in the sandbox with your community is essential, maintaining your original vision for entertainment that changes minds is equally important. He recommends having someone you can bounce ideas off to help maintain this balance, and remembers that all great things start small and are inherently iterative. Creating Emotionally Viable Products "We can't develop emotional connection by going through a list of features." Beyond minimum viable products, Patrick focuses on emotional viability - the hook that makes people truly care. Emotional connection cannot be built through feature lists but rather through compelling stories that capture people's imagination. When audiences engage with products outside of direct supervision, storytelling becomes the bridge that helps them discover new uses and applications. This creates a dance between product creators and their audience, leading to better product design. The Currency of Attention "Attention is the only currency - there's great wisdom in that." Patrick recognizes that in today's landscape, capturing and maintaining attention is the fundamental challenge. Since everyone is an audience member at different times, this perspective helps inform both strategy and tactics. Products must compete not just on functionality but on their ability to engage and maintain audience interest over time. As a recommended reading, Patrick suggests that we should read “Save The Cat! The Last Book on Screenwriting You'll Ever Need” to understand how to better tell stories about our products.  About Patrick James Lynch Patrick James Lynch is a filmmaker, media executive, and rare disease advocate. CEO of Believe Limited and founder of BloodStream Media, he uses his experience with hemophilia to drive award-winning storytelling, health advocacy, and mission-driven content that inspires and empowers rare and chronic disease communities worldwide. You can link with Patrick James Lynch on LinkedIn and follow Patrick James Lynch's work on his website.

On this episode, we explore the concept of shared decision making (SDM) in modern hemophilia care. With insights from experts Brendan Hayes and Dr. Cedric Hermans, the discussion explores the evolution of treatment options, patient education, and the collaborative decision-making process. The conversation also highlights the development and impact of the SDM tool by the World Federation of Hemophilia, emphasizing the importance of patient empowerment and ongoing education.   Guests: Kate Bazinsky Randy Curtis Debbie de la Riva Jackie Bottacari   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes:   #MyHemophiliaTruth. Real stories. Powered by Sanofi. It's time to consider the whole experience when it comes to managing hemophilia and change the conversation. Explore the full story at www.myhemophiliatruth.com   Emil's Story: Join Emil as he opens up about his journey living with hemophilia and the profound impact it has had on his mental health. He emphasizes how the effects of hemophilia extend beyond physical symptoms and underscores the importance of recognizing its emotional and psychological challenges. Emil also highlights the need for comprehensive care that supports both the mental and physical health of children and adults living with hemophilia.   Click here to watch his story: https://www.youtube.com/watch?v=9ljWxS57-ZA&list=PLmqBxf22n4lPMpAfLe4yv8haC6Ejcp1XL&index=2   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on X/Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter

CME credits: 1.25 Valid until: 30-04-2026 Claim your CME credit at https://reachmd.com/programs/cme/considering-the-promise-of-new-and-emerging-treatments-for-hemophilia-managing-thrombotic-risks/35552/ Stay ahead in hemophilia care with the new and emerging therapies transforming treatment approaches. The entire care team should be prepared to apply these innovations in a personalized way. This activity delivers key insights from the recent HTRS meeting along with foundational knowledge on novel therapies, presented in 5-minute modules for easy learning. *This content has not been endorsed by HTRS.

CME credits: 1.25 Valid until: 30-04-2026 Claim your CME credit at https://reachmd.com/programs/cme/the-impact-of-hemophilia-across-patients-lives/35547/ Stay ahead in hemophilia care with the new and emerging therapies transforming treatment approaches. The entire care team should be prepared to apply these innovations in a personalized way. This activity delivers key insights from the recent HTRS meeting along with foundational knowledge on novel therapies, presented in 5-minute modules for easy learning. *This content has not been endorsed by HTRS.

CME credits: 1.25 Valid until: 30-04-2026 Claim your CME credit at https://reachmd.com/programs/cme/an-overview-of-current-and-emerging-treatment-options-for-hemophilia-a-b/35546/ Stay ahead in hemophilia care with the new and emerging therapies transforming treatment approaches. The entire care team should be prepared to apply these innovations in a personalized way. This activity delivers key insights from the recent HTRS meeting along with foundational knowledge on novel therapies, presented in 5-minute modules for easy learning. *This content has not been endorsed by HTRS.

CME credits: 1.25 Valid until: 30-04-2026 Claim your CME credit at https://reachmd.com/programs/cme/multidisciplinary-team-collaboration-in-the-new-era-of-hemophilia-treatment/35554/ Stay ahead in hemophilia care with the new and emerging therapies transforming treatment approaches. The entire care team should be prepared to apply these innovations in a personalized way. This activity delivers key insights from the recent HTRS meeting along with foundational knowledge on novel therapies, presented in 5-minute modules for easy learning. *This content has not been endorsed by HTRS.

CME credits: 1.25 Valid until: 30-04-2026 Claim your CME credit at https://reachmd.com/programs/cme/considering-the-promise-of-new-and-emerging-treatments-for-hemophilia-the-need-for-iti/35551/ Stay ahead in hemophilia care with the new and emerging therapies transforming treatment approaches. The entire care team should be prepared to apply these innovations in a personalized way. This activity delivers key insights from the recent HTRS meeting along with foundational knowledge on novel therapies, presented in 5-minute modules for easy learning. *This content has not been endorsed by HTRS.

On this episode, we delve into the concept of shared decision making (SDM) in modern hemophilia care. With insights from experts Brendan Hayes and Dr. Cedric Hermans, the discussion explores the evolution of treatment options, patient education, and the collaborative decision-making process. The conversation also highlights the development and impact of the SDM tool by the World Federation of Hemophilia, emphasizing the importance of patient empowerment and ongoing education.   Guests: Brendan Hayes, MPH, CPH Cedric Hermans MD PhD FRCP   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes:   #MyHemophiliaTruth. Real stories. Powered by Sanofi. It's time to consider the whole experience when it comes to managing hemophilia and change the conversation. Explore the full story at www.myhemophiliatruth.com   Mark's Story Join Mark as he shares about his experience living with hemophilia, including the impact it had on building and maintaining relationships throughout grade school, college, dating, and raising his family. Mark also highlights how shared decision making with his healthcare providers, and strong family and caregiver support in those decisions, can make all the difference when it comes to managing the daily challenges of hemophilia.   Click here to watch his story: https://www.youtube.com/watch?v=tQ8oeM0zL24&list=PLmqBxf22n4lPMpAfLe4yv8haC6Ejcp1XL&index=8   Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on X/Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter   

New treatments for Prader-Willi Syndrome and hemophilia; FDA fast tracks a chlamydia vaccine candidate; over-the-counter test cleared for identifying chlamydia, gonorrhea and trichomoniasis; semaglutide improves walking ability in patients with peripheral artery disease; and Imfinzi combo therapy approved for MIBC.    

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Sanofi and Alnylam have received FDA approval for the first RNAi treatment for hemophilia, with the drug, Qfitlia, indicated for both hemophilia A and B. This approval is significant as it can be given regardless of the presence of neutralizing antibodies against clotting factors VIII or IX. However, the sudden departure of FDA director Peter Marks has caused uncertainty in the biopharma industry. In other news, Vertex has cut a diabetes asset but analysts remain optimistic about their phase III option. Lilly's RNA silencer has shown promising results in lowering a key cardiovascular biomarker. Trilink is offering custom guide RNAs for CRISPR workflow to accelerate therapy discoveries. Despite market challenges, the cell and gene therapy sector has seen a 30% investment surge. Companies like Amgen, Aldeyra, and Argenx are among those with upcoming FDA actions. Arbutus has announced layoffs, while big pharmas are pushing boundaries in radiopharmaceuticals. Michelle Werner of AltoRNA is focused on making better drugs. Safety questions are looming in Duchenne as Dyne and Wave plan FDA filings. There are job opportunities available in data management and program leadership within the biopharma industry.Moving on to other news, several big pharmaceutical companies such as Novartis, Bayer, AstraZeneca, Bristol Myers Squibb, and Eli Lilly are competing in the radiopharmaceuticals market, which is projected to be worth over $13 billion by 2033. The FDA is expected to announce decisions on therapies for dry eye disease soon. Michelle Werner, CEO of AllTrna, is focused on developing trna-based treatments for various diseases.Safety concerns are emerging in the Duchenne muscular dystrophy space as companies like Dyne and Wave plan FDA filings. The EU rejected Lilly's Alzheimer's drug Kisunla, Biontech's bispecific showed promise in treating SCLC patients, and Wave's duchenne exon-skipper reversed muscle damage in a mid-stage trial. Job opportunities within the biopharma industry were also highlighted for those interested.Thank you for tuning in to Pharma and Biotech daily - keeping you updated on all the latest news in the world of pharmaceuticals and biotechnology.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/AAPA information, and to apply for credit, please visit us at PeerView.com/MFK865. CME/AAPA credit will be available until March 10, 2026.Leveling the Standard of Care in Hemophilia A: Insights & Strategies for Developing Effective, Personalized Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/AAPA information, and to apply for credit, please visit us at PeerView.com/MFK865. CME/AAPA credit will be available until March 10, 2026.Leveling the Standard of Care in Hemophilia A: Insights & Strategies for Developing Effective, Personalized Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/AAPA information, and to apply for credit, please visit us at PeerView.com/MFK865. CME/AAPA credit will be available until March 10, 2026.Leveling the Standard of Care in Hemophilia A: Insights & Strategies for Developing Effective, Personalized Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/AAPA information, and to apply for credit, please visit us at PeerView.com/MFK865. CME/AAPA credit will be available until March 10, 2026.Leveling the Standard of Care in Hemophilia A: Insights & Strategies for Developing Effective, Personalized Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/AAPA information, and to apply for credit, please visit us at PeerView.com/MFK865. CME/AAPA credit will be available until March 10, 2026.Leveling the Standard of Care in Hemophilia A: Insights & Strategies for Developing Effective, Personalized Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/AAPA information, and to apply for credit, please visit us at PeerView.com/MFK865. CME/AAPA credit will be available until March 10, 2026.Leveling the Standard of Care in Hemophilia A: Insights & Strategies for Developing Effective, Personalized Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/AAPA information, and to apply for credit, please visit us at PeerView.com/MFK865. CME/AAPA credit will be available until March 10, 2026.Leveling the Standard of Care in Hemophilia A: Insights & Strategies for Developing Effective, Personalized Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/AAPA information, and to apply for credit, please visit us at PeerView.com/MFK865. CME/AAPA credit will be available until March 10, 2026.Leveling the Standard of Care in Hemophilia A: Insights & Strategies for Developing Effective, Personalized Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc.Disclosure information is available at the beginning of the video presentation.

Hemophilia is a rare bleeding disorder caused by a deficiency in clotting factors in the blood, which can cause permanent damage to joints and, in some cases, life threatening bleeding, both externally and internally. Today, people with hemophilia can live generally long, healthy lives, but in previous generations, the future wasn't so bright. In fact, less than a century ago the life expectancy for someone with hemophilia hovered around just 10 years. It doesn't receive much attention, and when it does, what you hear might not be all that accurate. In today's episode, we cover the history of hemophilia and the science behind treatments over the decades, including the devastating impact of the HIV/AIDS epidemic on hemophilia patients. We'll also clear up several misconceptions, including one that seems to show up a lot in popular culture: that hemophilia is a "royal disease.” Send us your science facts, news, or other stories for a chance to be featured on an upcoming Tiny Show and Tell Us bonus episode. And, while you're at it, subscribe to our newsletter!Link to the Tiny Show & Tell stories are here and here. All Tiny Matters transcripts and references are available here.See Privacy Policy at https://art19.com/privacy and California Privacy Notice at https://art19.com/privacy#do-not-sell-my-info.

In this week's episode we'll learn more about the significance of hypercalcemia in monoclonal gammopathy of undetermined significance, the role of neutrophil gelatinase-associated lipocalin in hemostasis, and the feasibility of combining CD19-targeted NK- or T-cell therapy with anti-CD19 monoclonal antibodies.Featured Articles:Approaching Hypercalcemia in Gammopathy of Undetermined Significance: Insights from the iStopMM study Deficiency of neutrophil gelatinase-associated lipocalin elicits Hemophilia-like bleeding and clotting disorder Anti-CD19 antibody cotreatment enhances serial killing activity of anti-CD19 CAR-T/-NK cells and reduces trogocytosis 

This episode of the VJHemOnc podcast covers updates in non-malignant hematological diseases from the 66th American Society of Hematology (ASH)... The post Post-ASH non-malignant highlights: updates in sickle cell disease, thalassemia, and hemophilia appeared first on VJHemOnc.

On this episode of the Global Hemophilia Report, we speak with guests who are each pioneering hemophilia advocacy in their respective regions. The discussion highlights the pivotal role of data and patient storytelling in driving systemic change and policy advancements. The episode emphasizes how global collaboration and technological innovations are paving the way for more equitable hemophilia care worldwide.    Guests: Harshal Kale, World Federation of Hemophilia Matthew Delaney, National Bleeding Disorders Foundation Megan Adediran, Hemophilia of Nigeria   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

Dr. Guy Young, Director of the Hemostasis and Thrombosis Program, Attending Physician in Hematology-Oncology, and Professor of Pediatrics at Keck School of Medicine at the University of Southern California (USC), is currently serving as co-chair of i3 Health's CME/NCPD activity, Practice-Changing Advances in the Management of Hemophilia. With new developments in the field occurring over recent months, Dr. Young sat down with us to share recent updates in the hemophilia treatment and management. Click the links below for the full activity! Online accredited CME/NCPD activity: https://i3health.com/course-information/practice-changing-advances-in-the-management-of-hemophilia Accredited CME/NCPD podcast: https://i3health.com/course-information/practice-changing-advances-in-the-management-of-hemophilia-podcast

Behind the Mystery is the only television series featuring rare, genetic diseases, and we have creators and producers Carri Levy and Molly Mager, to tell us all about it and how to watch. Also, from the Living with Hemophilia blog, Philippines residents Cza and Jared share their story about living with severe hemophilia B. All this and more!    Presenting Sponsor: Takeda, visit bleedingdisorders.com to learn more.   Show Notes: Subscribe: The BloodStream Podcast To learn more about our advocacy digital storytelling platform, Voices for Policy Change, head over to www.bleeding.org Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter

Clinical Clips in Hemophilia: Cutting-Edge Advances Presented at ASH 2024, will focus on the most exciting abstracts in hemophilia A and B that would be impactful for improved patient care. Dr. Guy Young will discuss the utility of key new evidence on replacement and non-replacement therapy to overcome current limitations and enable improved outcomes among patients.Launch Date: December 12, 2024Release Date: December 12, 2024Expiration Date: November 30, 2025FACULTYGuy Young, MDProfessor of PediatricsUniversity of Southern California Keck School of MedicineDirector, Hemostasis and Thrombosis CenterChildren's Hospital Los AngelesThis podcast provides accredited continuing education credits.  To receive your credit, please read the accreditation information provided at this link below prior to listening to this podcast.https://www.practicepointcme.com/CMEHome/clinical-clips-in-hemophilia-cutting-edge-advances-presented-at-ash-2024-1

Hemophilia A (HA) is an X-linked bleeding disorder caused by a mutation in the factor 8 (F8) gene that codes for FVIII coagulation protein. FVIII is naturally synthesized in the liver and the mutation results in the loss of function of FVIII protein which is critical in the blood clotting cascades. HA occurs is 1 to 5000 male births and it is estimated that there are a total of 3000 patients with HA in Canada. Currently, HA patients are treated with replacement therapy of the deficient factor. However, this approach is transient because of the short half-life of recombinant or plasma derived FVIII and is insufficient due to the formation of anti-drug antibodies. The alternative approach which is curative and promises a long-lasting expression of FVIII protein is gene therapy. For upcoming interviews check out the Grad Chat webpage on Queen’s University School of Graduate Studies & Postdoctoral Affairs website .

Supporting Georgians with Hemophilia, with Vic McCarty, Hemophilia of Georgia (North Fulton Business Radio, Episode 816) In this episode of North Fulton Business Radio, John Ray interviews Vic McCarty, Chief Community Engagement Officer with Hemophilia of Georgia. Vic discusses the organization’s extensive work in supporting individuals with blood clotting disorders, providing education, resources, and specialized care […] The post Supporting Georgians with Hemophilia, with Vic McCarty, Hemophilia of Georgia appeared first on Business RadioX ®.

Supporting Georgians with Hemophilia, with Vic McCarty, Hemophilia of Georgia (North Fulton Business Radio, Episode 816) In this episode of North Fulton Business Radio, John Ray interviews Vic McCarty, Chief Community Engagement Officer with Hemophilia of Georgia. Vic discusses the organization’s extensive work in supporting individuals with blood clotting disorders, providing education, resources, and specialized care […]

In the next podcast episode, we will continue our talk on hemophilia. Specifically the treatment of hemophilia. We'll discuss the different types of therapy available, from traditional factor replacement to emerging gene-techniques. Join Dr. Niket Sonpal as he helps us manage hemophilia patients.

Have you ever wondered about the history hemophilia? In this episode, we will dive into the world of hemophilia, a hereditary bleeding disorder that affects millions worldwide. We'll explore the basics of this condition, including genetic causes and how it impacts individuals. Join Dr. Niket Sonpal for another genetic blood disorder.

In this episode, we review the high-yield topic of ⁠Hemophilia⁠⁠⁠⁠⁠⁠ ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠from the Heme section. Follow ⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠Medbullets⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠⁠ on social media: Facebook: www.facebook.com/medbullets Instagram: www.instagram.com/medbulletsofficial Twitter: www.twitter.com/medbullets Linkedin: https://www.linkedin.com/company/medbullets

On this episode we talk with experts Dr. Amy Shapiro, Dr. Maria Elisa Mancuso, Dr. Steve Pipe, Dr. Johnny Mahlangu, and Dr. Lynn Malec to delve into the ongoing evolution of hemophilia therapies. The discussion highlights recent advancements in treatments such as emicizumab, extended half-life factor VIII therapies, and investigates medications in clinical trials like Concizumab, Marstacimab, and Fitusiran. The episode also reflects on the role of treatment individualization and the need for more inclusive research data.   Contributors: Johnny Mahlangu, MBBCh, MMed, FCPath Lynn Malec, MD, MSc Elisa Mancuso, MD Steven Pipe, MD Amy Shapiro, MD   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch   Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report   Show Notes: Connect with the Global Hemophilia Report Global Hemophilia Report on LinkedIn Global Hemophilia Report on Twitter Global Hemophilia Report on Facebook   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

Patrick has an exclusive conversation with the filmmakers of What We Wished We Could Be, a story about a couple who must sacrifice their dreams in order to confront the consequences of the contaminated blood scandal. Patrick sits down in-studio with screenwriter, Jenna-Louise Hawkins; Director, Luke Shelley; and producer Sophie Stacy about the challenges of making the film, Jenna's personal connection to the UK contamination scandal, and how storytelling can honor the heartbreak, courage and sacrifice of the victims of the blood scandal, and their loved ones. Plus, the second installment of Jack Bridge's interview with Luke Pembroke in I'm Fine segment.    Presenting Sponsor: Takeda, visit bleedingdisorders.com to learn more.   Show Notes: Subscribe: The BloodStream Podcast   Visit BEQVEZ.com to learn more about gene therapy, sign up for additional information, and listen to real patients who are now living differently.   To learn more about Miranda and others in the hemophilia community, please visit www.treathemA.com   I'm Fine is presented by @SanofiUS    What We Wished We Could Be film: Website - Instagram - Trailer   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter

This week's episode will be repeating our episode on hemophilias with some updates. We will go over all high yield facts regarding both hemophilia A and B  for your boards and end with a little bit about acquired hemophilias. 

Hemophilia is a bleeding disorder resulting from defects in factors of the coagulation cascade. There are two primary types of hemophilia: A and B. Both types of hemophilia are rare, with only a combined frequency of about 1 in 5000 live births. Of the two types, hemophilia A is about four times more common. In the intrinsic arm of the coagulation cascade, factor VIII serves as a cofactor for factor IX. They cooperate to activate factor X, leading to the formation of fibrin. After listening to this AudioBrick, you should be able to: Compare and contrast the underlying defect in hemophilia A and B. Describe the inheritance pattern and relative incidence of hemophilia A and B. Describe the clinical features of hemophilia A and B. Explain how hemophilia A and B are diagnosed. List and briefly describe other factor deficiencies. You can also check out the original brick on Hemophilia from our Hematology collection, which is available for free. Learn more about Rx Bricks by signing up for a free USMLE-Rx account: www.usmle-rx.com You will get 5 days of full access to our Rx360+ program, including nearly 800 Rx Bricks.  After the 5-day period, you will still be able to access over 150 free bricks, including the entire collections for General Microbiology and Cellular and Molecular Biology. *** If you enjoyed this episode, we'd love for you to leave a review on Apple Podcasts.  It helps with our visibility, and the more med students (or future med students) listen to the podcast, the more we can provide to the future physicians of the world. Follow USMLE-Rx at: Facebook: www.facebook.com/usmlerx Blog: www.firstaidteam.com Twitter: https://twitter.com/firstaidteam Instagram: https://www.instagram.com/firstaidteam/ YouTube: www.youtube.com/USMLERX Learn how you can access over 150 of our bricks for FREE: https://usmlerx.wpengine.com/free-bricks/

Mild hemophilia truly does matter and we're with Shellye Horowitz, formally with HFA, to hear about HFA's Mild Matters program. Plus the final Elite Athletes segment with Paul McLaughlin and I'm Fine with Luke Pembroke. Show Notes: Subscribe: The BloodStream Podcast   Presenting Sponsor: Takeda, visit bleedingdisorders.com to learn more.   I'm Fine and Elite Athletes with Hemophilia are presented by @SanofiUS    Mild Matters Program   On the Shoulders of Giants Film Watch the movie and host a screening: ontheshouldersfilm.com    Beyond our Blood Watch the short films and download the facilitation guide: beyondourblood.com   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter

Hear about the premiere of the On the Shoulders of Giants film from HFA Symposium with Patrick and Amy. Plus the I'm Fine segment is back with Luke Pembroke and Hazri Aris is featured on the latest Elite Athletes segment.  Show Notes: Subscribe: The BloodStream Podcast   Presenting Sponsor: Takeda, visit bleedingdisorders.com to learn more. On the Shoulders of Giants Film Watch the movie and host a screening: ontheshouldersfilm.com    Beyond our Blood Watch the short films and download the facilitation guide: beyondourblood.com   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter

With its potential to expand the horizons of what's possible for patients, CRISPR is the new darling of biotech. Reaching an important milestone in 2023 with the FDA approval of a sickle cell treatment using CRISPR, the technology is poised to break new barriers for treating patients in the near future. In this episode we talk with two companies working together on the next generation of CRISPR: editing cells within the body.  We discuss the progress and the challenge in making this breakthrough a reality. Follow us on LinkedIn, X, Facebook and Instagram. Visit us at https://www.bio.org/

A 5-year-old child and his parents boarded a plane heading for a new life. They were Syrian refugees, who fled conflict in their country and then lived a difficult life in a refugee camp. Now they were heading to Canada. But soon after arriving, there's a problem. The child has hemophilia and due to hemophilia complications, a minor injury while traveling became a major concern. Add to this a language barrier and trying to understand a new culture. Dr. Robert Klaassen, a pediatric hematologist and lead of the Comprehensive Hemophilia Care Clinic at Children's Hospital of Eastern Ontario in Ottawa, Canada, shares his experience working with a family who overcame incredible barriers and the lessons learned along the way.

It was supposed to be a simple, low-risk procedure, but for this 61-year-old patient with undiagnosed hemophilia, undergoing a lithotripsy for kidney stones proved to be anything but. The patient was born in the 1950s in Taiwan, when many people in the country had never heard of the disease. So, despite signs throughout his life, the patient's hemophilia diagnosis evaded him for decades. Dr. Yeu-Chin Chen, a hematologist at the Tri Service General Hospital's Hemophilia Care and Research Center in Taipei, Taiwan, shares this patient's journey and how doctors should be on the lookout for signs of hemophilia, while understanding that symptoms can vary from patient to patient.

A young child living with severe hemophilia is adopted by a family in the United States. Prior to his adoption, due to a lack of resources and other challenges, his hemophilia was not properly managed. Dr. Meera Chitlur, a pediatric hematologist and the director of the Hemophilia Treatment Center at the Children's Hospital of Michigan in Detroit, has treated this patient since he first came to the U.S. As he grew older, like many children, he wanted to play sports. But for people with hemophilia, participating in sports brings great risk. Dr. Chitlur shares how together with the patient and his family, they navigated the challenges of growing up with hemophilia and how new treatment options for pediatric hemophilia have opened up a whole new world for kids living with the disease.

About 40 years ago a mother brought her 6-month-old child into the hospital. He was covered in bruises. Dr. Victor Blanchette, a pediatric hematologist at the Hospital for Sick Children in Toronto, Canada, met the patient that day and, following a severe hemophilia diagnosis, has treated the patient ever since. Dr. Blanchette recounts how during the patient's childhood in the 80s, the approach to treating hemophilia was reactive, not proactive. This meant that normal childhood activities could lead to devastating bleeds. During this episode, we walk through the history of hemophilia and how its evolution toward preventive care has had an incredible impact on this patient and so many others.

A 30-year-old was in labor with her first child. Everything was going to plan … until it wasn't. Dr. Azusa Nagao, a hematologist at Ogikubo Hospital in Tokyo, Japan, shares a case that illustrates how historically it was thought that women and people assigned female at birth could only be carriers of the disease, not have the disease themselves. This misunderstanding of hemophilia in women has led to women going undiagnosed and untreated with severe repercussions. For the patient in this story, it meant a frightening and dangerous birth experience that put her and her child at risk. Dr. Nagao also outlines efforts to educate patients and physicians about hemophilia, what to look for, and how to treat it.

A patient was experiencing severe knee pain. Unfortunately, this wasn't anything new. For decades, he'd been suffering from joint disease, caused by severe bleeding in his knees from a rare blood disorder — hemophilia A. The patient was born in the 1960s, a time when the life expectancy for patients with hemophilia was only 10 years. But as Dr. Annette Von Drygalski, a board certified hematologist and the director of the Hemophilia and Thrombosis Treatment Centre at the University of California, San Diego, explains, advances in hemophilia treatment throughout this patient's life allowed him — and many others like him — to live a longer, richer life.

Go inside the minds of doctors who specialize in hemophilia — a rare, inherited bleeding disorder that once meant possibly not surviving past the age of 20. This season, we'll explore the medical milestones that enable patients today to live longer, richer lives and examine the challenges yet to be tackled.