Podcasts about hemophilia

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 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. 

This week's VJHemOnc podcast brings you updates in hemophilia from the 32nd Congress of the International Society on Thrombosis and... The post Hemophilia updates from ISTH 2024: ongoing clinical trials, emerging gene therapies & remaining challenges appeared first on VJHemOnc.

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/

In this podcast, hear expert perspectives on practice-changing advances in the treatment and personalized management of patients with hemophilia from Dr. Guy Young, Director of the Hemostasis and Thrombosis Program, Attending Physician in Hematology/Oncology, and Professor of Pediatrics at Keck School of Medicine of USC; and Dr. Jennifer Davila, Co-Director of the Hemophilia Treatment Center at Montefiore and Associate Professor of Pediatrics at The Children's Hospital at Montefiore. Listen to the episode now! Click here to claim your free CME/NCPD credit for this activity: https://bit.ly/4eQSbaL

In this week's episode we'll learn about fitusiran prophylaxis in patients with hemophilia A or B, with or without inhibitors. Next we'll hear about new findings that heterozygous germline variants in the NBN gene that are linked to increased risk of B-cell acute lymphoblastic leukemia in children. Finally, we'll explore new insights on the histone demethylase PHF8, which has been identified as a master regulator of cell-intrinsic immune responses in acute myeloid leukemia. Featured Articles:Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trialGermline genetic NBN variation and predisposition to B-cell acute lymphoblastic leukemia in children Epigenetic control over the cell-intrinsic immune response antagonizes self-renewal in acute myeloid leukemia

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

            For medical writing assistance, please visit nascentmc.com learnAMAstyle.com for the AI in Medical Writing and Editing: Blueprint for Success in a Changing World course to assist with implementing AI in medical writing.  •    Imdelltra for EC-SCLC: The FDA granted accelerated approval for tarlatamab-dlle (Imdelltra) for adults with extensive-stage small cell lung cancer (ES-SCLC) who progressed after platinum-based chemotherapy. Small cell lung cancer, accounting for 10-15% of all lung cancers, is aggressive with a median survival of 12 months post-therapy. Imdelltra, a first-in-class BiTE therapy targeting DLL3, showed a 40% objective response rate and a median overall survival of 14.3 months in the phase 2 DeLLphi-301 trial. •    Breyanzi for R/R LL: The FDA granted accelerated approval for lisocabtagene maraleucel (Breyanzi) for adults with relapsed or refractory follicular lymphoma after two or more systemic therapies. This CAR T-cell therapy, already approved for other B-cell malignancies, genetically modifies T cells to target tumor antigens. In the TRANSCEND FL trial, Breyanzi achieved a 95.7% overall response rate in 94 patients, with the median response duration not reached after 16.8 months. •    SNB-101 for SCLC: The FDA granted fast-track designation to SNB-101 for small cell lung cancer (SCLC). SNB-101, a polymer nanoparticle formulation of the antineoplastic agent SN-38, enhances lung-specific delivery and efficacy. Early data indicates improved tolerability and efficacy, with global phase 2 trials planned for various solid tumors including colon and gastric cancers, led by SN BioScience. •    Upstaza for AADC Deficiency: The FDA accepted the Biologics License Application for eladocagene exuparvovec (Upstaza), a gene therapy for aromatic L–amino acid decarboxylase (AADC) deficiency, with a Priority Review and a target date of November 13, 2024. Upstaza delivers the human DDC gene to the putamen, increasing AADC enzyme levels to restore dopamine production, showing significant neurological improvements in clinical trials. Approval was granted to PTC Therapeutics, Inc. •    Altuviiio for Hemophilia A Label Update: The FDA approved an updated label for Altuviiio (antihemophilic factor, recombinant), including phase 3 XTEND-Kids trial results. This high-sustained factor VIII replacement therapy, initially approved in 2023, offers prolonged bleed protection for pediatric patients under 12 years with hemophilia A. The updated label confirms Altuviiio's safety and effectiveness for both routine and on-demand treatment. •    Self-Collected Sample for Cervical Cancer Screening: The FDA approved the BD Onclarity™ HPV Assay for self-collected vaginal specimens for HPV testing when traditional cervical samples are not available. This assay, identifying six HPV strains, enhances screening access, especially for underserved populations with higher cervical cancer rates. Approval was granted to Becton Dickinson. •    Clinolipid Parenteral Nutrition: The FDA approved Clinolipid (Lipid Injectable Emulsion) for pediatric patients, including preterm and term neonates. Clinolipid, a mixed oil lipid emulsion providing essential fatty acids and calories, has been available for adults since 2019 and is now approved for all ages. Approval was granted to Baxter International Inc.

ASGCT Board of Directors member Lindsey George, MD, (Children's Hospital of Philadelphia) discusses the dramatic advancements in available treatment options for patients with hemophilia and the responsibility of scientists and clinicians in guiding patients through these once-in-a-lifetime decisions. Host: Lynnea Olivarez, incoming chair of the ASGCT Communications CommitteeShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

VIsit Nascentmc.com for medical writing assistance. Visit learnamastyle.com for freebies on medical writing and editing and ChatGPT. - Fasenra Pediatric Asthma Expansion: No details provided for this update. - Beqvez for Hemophilia B: FDA approved gene therapy, Beqvez, for adults with moderate to severe hemophilia B, which enables the production of clotting protein factor IX. This one-time treatment by Pfizer, derived from Spark Therapeutics, aims to replace frequent infusion therapies, demonstrating superior efficacy in a late-stage trial. - Anktiva for Bladder Cancer: Anktiva received FDA approval for treating BCG-unresponsive non-muscle invasive bladder cancer, enhancing NK and T cell proliferation. Based on a trial with 77 patients, it showed a 62% complete response rate, surpassing international clinical benchmarks, leading to its designation as a breakthrough therapy. - Pivya for UTI: FDA approved pivmecillinam (Pivya) for treating uncomplicated urinary tract infections caused by specific bacterial strains. This marks the first new antibiotic for such infections in the U.S. in over 20 years, backed by effective outcomes in three clinical trials. - Tovorafenib for Pediatric Low-Grade Glioma: Tovorafenib was approved for pediatric low-grade glioma patients with specific BRAF alterations, showing a 67% response rate in the FIREFLY-1 trial. It has been designated for accelerated approval due to its potential in treating these brain tumors. - Entyvio Maintenance for Crohn's Disease: Vedolizumab (Entyvio) received approval for subcutaneous administration as Crohn's disease maintenance therapy after initial intravenous induction. Supported by the VISIBLE 2 Study, it proved effective in maintaining clinical remission at 52 weeks. - Alecensa for NSCLC: Alecensa was approved as an adjuvant treatment post-tumor resection for ALK-positive non-small cell lung cancer. In the ALINA trial, it significantly extended disease-free survival compared to chemotherapy, particularly in early-stage patients. - SPG601 for Fragile X Syndrome: The FDA cleared SPG601 for a phase 2a trial in Fragile X Syndrome, addressing synaptic function through BK channel activation. This marks an advance for treating the core symptoms of the most common inherited intellectual disability. - Lumisight for Visualizing Breast Cancer: The FDA approved Lumisight and the Lumicell Direct Visualization System for use during lumpectomy surgeries to detect residual cancer tissues. This system, shown in the INSITE trial, improves surgical outcomes by reducing the need for second operations. VIsit Nascentmc.com for medical writing assistance. Visit learnamastyle.com for freebies on medical writing and editing and ChatGPT.

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

On this episode of the Global Hemophilia Report, host Patrick James Lynch is on location in Madrid, Spain for the WFH 2024 World Congress. Patrick talks about the history of World Hemophilia Day and talks with some of the attendees at the congress. Contributors: Hazri Aris Johnny Mahlangu Rebecca, Hematology Resident   Senior Advisor: Donna DiMichele, MD   Hosted & Written by: Patrick James Lynch Featured Advertiser: Sanofi   Subscribe to the Global Hemophilia Report Show Notes: Presenting Sponsor: Sanofi   Subscribe to the Global Hemophilia Report   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

On 18 April 2024, World Hemophilia Day was observed in the Kurdistan region at the GIN Blood Centre in Duhok, Iraq. Many children with blood diseases enjoyed fun activities and were handed gifts and toys. Dr Omid Adnan, a specialist in blood disease treatment at the centre, discussed its significance with Naseem Sadiq, who filed this report.

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.

In this episode of the podcast we talk with Kristina Robinson, a patient advocate and mother whose son, Axel, was diagnosed with hemophilia A when he was 10 months old. That's a rare bleeding disorder, sometimes called “classic hemophilia,” that is characterized by excessive bleeding from cuts, unexplained bruising, joint swelling and more. Since her son was diagnosed, Kristina has been his #1 champion, advocating for hemophilia awareness in her home state and beyond. Keep up with Kristina and Axel on Instagram! Learn more about hemophilia and ways that you can get involved at the New England Hemophilia Association! Editor's Note: Chronic conditions and rare diseases don't discriminate. Patient Worthy and our partners are interested in amplifying the voices of those from all identities and backgrounds. If you have a story to share, learn more about how your voice can help spread awareness and inspire individuals from all walks of life here: https://shorturl.at/sPV02

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.

HFA CEO Dan Kelsey joins Patrick and Amy to respond to recent organizational changes, followed by Blood Brother, Carl Weixler, who shares a bit about HFA's history, mission, and current state. Also, a tribute to Rare Disease Day, led by voices from BloodStream Media. Show Notes: Subscribe: The BloodStream Podcast   Presenting Sponsor: Takeda, visit bleedingdisorders.com to learn more.   Check out the Everyday Life Foundation hosting Bombardier Blood Everyday Life Foundation Legislative Asks   This Rare Disease Day segment is brought to you by Genentech. Genentech has several resources to help caregivers navigate hemophilia including stories from other members in the community. Please visit www.thecommunityhelps.com to learn more.   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter   

We've got a terrific interview with Kathy MacKay and Dana Kuhn about the efforts the Committee of Ten Thousand (COTT) is taking to preserve documents from the tainted blood tragedy. And another Elite Athletes segment featuring blood brother and cyclist, Giovanny Pernudi. Oh …and Patrick had a bath bleed.  Show Notes: Subscribe: The BloodStream Podcast To learn more about the Committee of Ten Thousand and to get involved, visit www.COTT1.org Presenting Sponsor: Takeda, visit bleedingdisorders.com to learn more.   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on Twitter