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  • How the Patient Perspective Led to a Hemophilia Medical Breakthrough

    How the Patient Perspective Led to a Hemophilia Medical Breakthrough
    2026/07/08
    Please see https://www.pfi.sr/HYMPAVZI_PI for Full PI. #HYMPAVZISponsored William S. Somers, Ph.D., was a biotherapeutics research and development leader with more than 30 years of experience across biotechnology and pharmaceutical organizations, including Pfizer. As a scientist living with hemophilia, Dr. Somers played a key role in driving the patient-centric and purposeful design of HYMPAVZI (marstacimab-hncq). This episode will explore how the patient perspective influenced the scientific journey, with a special focus on Will's research and why it was personally important for him to bring HYMPAVZI to people with hemophilia. HYMPAVZI may cause serious side effects, including blood clots (thromboembolic events). HYMPAVZI may increase the risk for your blood to clot in blood vessels in your arm, leg, lung, or head, which can be life-threatening. To learn more about this risk and additional safety information visit HYMPAVZI.com. This post is directed to a U.S. audience and may not be suitable outside of the U.S. This video is intended to be viewed as it was originally produced in partnership with Pfizer. Remember, this content is for educational purposes only and does not replace discussions with a healthcare provider. PP-HYM-USA-1267 Show Notes: For safety information visit: https://labeling.pfizer.com/ShowLabeling.aspx?id=20916§ion=PPI For information on how to navigate your treatment journey, visit the Shared Decision-making Guide here. Listen and subscribe where you get your podcasts! #Hemophilia IMPORTANT SAFETY INFORMATION Important: Before you start using HYMPAVZI, it is very important to talk to your healthcare provider about using factor VIII and factor IX products or "bypassing agents" (products that help blood clot but work in a different way than HYMPAVZI). Your healthcare provider may prescribe factor VIII or factor IX medicines or bypassing agents to treat episodes of breakthrough bleeding during your treatment with HYMPAVZI. Carefully follow your healthcare provider's instructions regarding when to use these medicines and the prescribed dose during your treatment with HYMPAVZI. Do not use additional doses of HYMPAVZI to treat breakthrough bleeds. Before using HYMPAVZI, tell your healthcare provider about all of your medical conditions, including if you: have a planned surgery. Your healthcare provider may stop treatment with HYMPAVZI before your surgery. Talk to your healthcare provider about when to stop using HYMPAVZI and when to start it again if you have a planned surgery. have a severe short-term (acute) illness such as an infection or injury. have been told that you have a risk for blood clots. are pregnant or plan to become pregnant. HYMPAVZI may harm your unborn baby. Females who are able to become pregnant: Your healthcare provider will do a pregnancy test before you start your treatment with HYMPAVZI. Use effective birth control (contraception) during treatment with HYMPAVZI and for at least 2 months after the last dose of HYMPAVZI. Tell your healthcare provider right away if you become pregnant or think that you may be pregnant during treatment with HYMPAVZI. are breastfeeding or plan to breastfeed. It is not known if HYMPAVZI passes into your breast milk. Tell your healthcare provider about all the medicines you take, including prescription medicines, over-the-counter medicines, vitamins, and herbal supplements. If you inject too much HYMPAVZI, call your healthcare provider or the Poison Help Line at 1-800-222-1222 or go to the nearest hospital emergency room right away. What are the possible side effects of HYMPAVZI? HYMPAVZI may cause serious side effects, including: blood clots (thromboembolic events). HYMPAVZI may increase the risk for your blood to clot in blood vessels in your arm, leg, lung, or head, which can be life-threatening. Blood clots have happened in people using HYMPAVZI. You may have an increased risk of blood clots if you have certain risk factors. Stop using HYMPAVZI and get medical help right away if you develop any of these signs or symptoms of blood clots: swelling or pain in your arms or legs redness or discoloration in your arms or legs shortness of breath pain in chest or upper back fast heart rate cough up blood feeling faint headache numbness in your face eye pain or swelling trouble seeing allergic reactions. HYMPAVZI may cause allergic reactions, including rash and itching. Stop using HYMPAVZI and get medical help right away if you develop any of the following symptoms of a severe allergic reaction: swelling of your face, lips, mouth, or tongue trouble breathing wheezing dizziness or fainting fast heartbeat or pounding in your chest sweating The most common side effects of HYMPAVZI include: swelling, hardening, redness, bruising, bleeding, and pain at injection site headache fever joint pain diarrhea itching rash These are not all the possible side effects of HYMPAVZI. Call your doctor for ...
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    21 分
  • Plasma donors, RARE BLOOD, and advocacy wins

    Plasma donors, RARE BLOOD, and advocacy wins
    2026/04/24

    On this episode of BloodStream—our plasma donation series continues with a powerful second installment featuring donor Hannah and the story behind what it really means to give.

    We've also got a packed lineup: a look at momentum building at the WFH World Congress, policy progress led by State Senator Joseph Addabbo supporting families with rare disorders, and new momentum in storytelling as Sanofi backs additional documentaries spotlighting rare blood conditions.

    Plus, we dig into the bigger picture—how bleeding disorders impact entire families, the role creativity plays when navigating physical limitations, and the isolation that can come with not knowing someone who shares your experience.

    And don't miss a spotlight on The Red Ink Project and their virtual screening of Dismissed, a film bringing long-overdue visibility to women and girls with bleeding disorders.

    All that and more—this is BloodStream.

    Show Notes:

    NY State Senator Joseph Addabbo steps up for bleeding disorders

    Sanofi backs 2 more documentary films about rare blood disorders

    theredinkproject.org

    Presenting Sponsor:

    Takeda, visit bleedingdisorders.com to learn more.

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    24 分
  • What happened to HEMGENIX!? An update on gene therapy.

    What happened to HEMGENIX!? An update on gene therapy.
    2026/04/10

    CSL Behring just announced a global "stockout" of Hemgenix—what's happening, and what does it mean for patients? On this episode of BloodStream Podcast, we break down the news, launch our mini-series on plasma donation, and hear from Thomas Savage in our I'm Fine segment, sharing the highs, the lows, and the reality of living with a severe bleeding disorder.

    Pain, perseverance, and the lessons learned along the way give a rare, unfiltered glimpse into life behind the headlines—reminding us all that every treatment, every donation, and every day matters.

    Show Notes:

    I'm Fine is presented by @SanofiUS

    #bloodstreammedia #raredisease #bleedingdisorders #podcast #chronicdisease #hemophiliacommunity #advocacy

    Presenting Sponsor:

    Takeda, visit bleedingdisorders.com to learn more.



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    34 分
  • Understanding Hemophilia A & B with Inhibitors: SEVENFACT in Focus

    Understanding Hemophilia A & B with Inhibitors: SEVENFACT in Focus
    2026/04/03

    In this special partnered episode of the BloodStream Podcast, hosts Patrick James Lynch and Amy Board speak with hematologist Dr. Tami Singleton about hemophilia A and B with inhibitors, a complex condition that changes how bleeding episodes are treated. Dr. Singleton explains what inhibitors are, why they develop, and how they impact clotting and everyday life for patients and families. The conversation also explores SEVENFACT, a recombinant factor VIIa bypassing agent, including how it works and what patients should know about dosing and treatment planning.


    Click here to learn more about SEVENFACT.
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    41 分
  • 100 Years of vWD w/ Dr. Nathan Connell

    100 Years of vWD w/ Dr. Nathan Connell
    2026/03/27

    This year marks 100 years since von Willebrand disease (vWD) was first described, and on this episode of BloodStream we take a closer look at how far the field has come.

    Our beloved hosts, Patrick and Amy happily welcome Dr. Nathan Connell, hematologist and leading expert in bleeding disorders, for a conversation about the past, present, and future of vWD. Together they explore how our understanding of the condition has evolved since its discovery, the challenges that remain in diagnosis and care, and why awareness still matters for the millions of people worldwide living with vWD.

    From early clinical observations to modern treatment approaches, Dr. Connell reflects on the milestones that have shaped care over the last century—and what the next hundred years could hold.

    Plus, a preview of what's coming next on BloodStream as we approach World Hemophilia Day.

    Show Notes:

    Presenting Sponsor:

    Takeda, visit bleedingdisorders.com to learn more.

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    33 分
  • Psychosocial support and bye bye, BioMarin

    Psychosocial support and bye bye, BioMarin
    2026/03/13

    Bleeding disorders aren't just medical conditions — they're lived experiences shaped by resilience, community, and support. On this episode of BloodStream Podcast, Patrick James Lynch sits down with the WFH working group to explore why psychosocial care matters just as much as medicine and how human connections help people thrive.

    Rocky Williams joins to share the power of positivity and what it truly means to be more than "fine," inspiring the bleeding disorders community to dream bigger, aim higher, and embrace life fully. We also break down a major shift in gene therapy: BioMarin's decision to pull Roctavian from the market, what it means for patients, and why preserving critical clinical data is essential for ongoing research and safety.

    Tune in for a conversation that blends science, mindset, and community — because living with bleeding disorders is about more than treatments, it's about life itself.

    Show Notes:

    BioMarin pulls hemophilia gene therapy Roctavian

    WFH Gene Therapy Registry

    LevelsMatter.com

    Presenting Sponsor:

    Takeda, visit bleedingdisorders.com to learn more.

    I'm Fine is presented by @SanofiUS

    #bloodstreammedia #raredisease #bleedingdisorders #podcast #chronicdisease #hemophiliacommunity #advocacy

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    48 分
  • Jeff & Barb with Connie Montgomery!

    Jeff & Barb with Connie Montgomery!
    2026/02/27

    On this episode of BloodStream, we're thrilled to sit down with Connie Montgomery alongside Jeff and Barb for a warm, wide-ranging conversation grounded in lived experience and deep community roots. Together, they reflect on the moments that shape advocacy, the evolution of the bleeding disorders space, and the power of staying connected to one another.

    Plus, a nod to Rare Disease Day, a bit of BloodStream banter, and a preview of what's ahead.

    Tune in, share with your community, and as always—take self-care of yourself.

    Presenting Sponsor:

    Takeda, visit bleedingdisorders.com to learn more.

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    31 分
  • A preview of 2026 World Congress with Dr. Glenn Pierce

    A preview of 2026 World Congress with Dr. Glenn Pierce
    2026/02/20

    On this episode of BloodStream, we get a sneak peek at the 2026 World Congress with Dr. Glenn Pierce, Vice President, Medical at the World Federation of Hemophilia. What conversations are shaping the agenda? Where is the science heading? And how does a global convening like World Congress moves the needle on access, equity, and innovation for people living with bleeding disorders? Dr. Pierce shares why this gathering still matters — and what the community should be watching as 2026 approaches.

    Plus, in I'm Fine, we sit down with Anthony, a performer and educator living with a bleeding disorder. From invisible disability to grieving the life you imagined, it's a candid conversation about ambition, identity, and learning to stop blaming yourself for what you can't control.

    Global outlook. Personal reckoning. Classic BloodStream.

    Presenting Sponsor:

    Takeda, visit bleedingdisorders.com to learn more.

    I'm Fine is presented by @SanofiUS

    Subscribe:

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    33 分