BloodStream

BloodStream is an entertaining and informational podcast for the bleeding disorders community. Each week, hemophilia patient Patrick James Lynch and former non-profit director Amy Board are joined by advocacy leaders, medical experts, and other guests to discuss the latest news, scientific advancements, policy initiatives, and so much more. Subscribe wherever you listen, or search BloodStream Podcast on YouTube to catch every episode there. Produced by Believe Limited. Presenting sponsor: Takeda.

  1. How the Patient Perspective Led to a Hemophilia Medical Breakthrough

    4D AGO

    How the Patient Perspective Led to a Hemophilia Medical Breakthrough

    Please see https://www.pfi.sr/HYMPAVZI_PI  for Full PI​ #HYMPAVZISponsored William S. Somers, Ph.D. is 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. To learn more and for 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 is not intended to replace discussions with a healthcare provider.​ PP-HYM-USA-1071​ Show Notes:​ For prescribing information visit pfi.sr/HYMPAVZI_PI​ 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 HIGHLIGHTS OF PRESCRIBING INFORMATION These highlights do not include all the information needed to use HYMPAVZI safely and effectively. See full prescribing information for HYMPAVZI. HYMPAVZI (marstacimab-hncq) injection, for subcutaneous use Initial U.S. Approval: 2024 INDICATIONS AND USAGE HYMPAVZI is a tissue factor pathway inhibitor (TFPI) antagonist indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with: hemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors, or hemophilia B (congenital factor IX deficiency) without factor IX inhibitors. DOSAGE AND ADMINISTRATION See Full Prescribing Information for important dosing and administration instructions.  The recommended dosage of HYMPAVZI is: Loading dose: 300 mg (two 150 mg injections) by subcutaneous injection Maintenance dose: One week after the loading dose, initiate maintenance dosing of 150 mg every week by subcutaneous injection on the same day each week, at any time of day. Dose adjustment to 300 mg subcutaneous injection weekly can be considered. Factor VIII and factor IX products can be administered for the treatment of breakthrough bleeds in patients receiving HYMPAVZI. Do not use additional doses of HYMPAVZI to treat breakthrough bleeds.  Temporarily discontinue HYMPAVZI before major surgery.  DOSAGE FORMS AND STRENGTHS Injection: 150 mg/mL in a single-dose prefilled syringe  Injection: 150 mg/mL in a single-dose prefilled pen  CONTRAINDICATIONS None. WARNINGS AND PRECAUTIONS Thromboembolic Events: Thromboembolic events may occur. Interrupt HYMPAVZI prophylaxis if symptoms occur. Hypersensitivity: Hypersensitivity reactions may occur. In the event of a severe allergic reaction, discontinue HYMPAVZI. Embryofetal Toxicity: May cause fetal harm. Advise females of reproductive potential of the potential risk to the fetus and to use effective contraception. ADVERSE REACTIONS Adverse reactions reported in ≥3% of HYMPAVZI-treated patients were injection site reaction, headache, and pruritus.  To report SUSPECTED ADVERSE REACTIONS, contact Pfizer Inc. at 1-800-438-1985 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. See 17 for PATIENT COUNSELING INFORMATION and FDA-approved patient labeling. Revised: 12/2025

    21 min
  2. Psychosocial support and bye bye, BioMarin

    MAR 13

    Psychosocial support and bye bye, BioMarin

    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   Subscribe: The BloodStream Podcast   Connect with BloodStream Media: BloodStreamMedia.com BloodStream on Facebook  BloodStream on X/Twitter  BloodStream on Instagram BloodStream on LinkedIn BloodStream on TikTok

    48 min
4.9
out of 5
43 Ratings

About

BloodStream is an entertaining and informational podcast for the bleeding disorders community. Each week, hemophilia patient Patrick James Lynch and former non-profit director Amy Board are joined by advocacy leaders, medical experts, and other guests to discuss the latest news, scientific advancements, policy initiatives, and so much more. Subscribe wherever you listen, or search BloodStream Podcast on YouTube to catch every episode there. Produced by Believe Limited. Presenting sponsor: Takeda.

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