Patrick and Amy sit down to hear her story—how she first got involved in the bleeding disorders community back in 2003, and how patient advocacy became her passion. Along the way, they share laughs about their unexpected pasts as actors and the moments that shaped their paths.

Also on this episode of BloodStream, our hosts also look back on the year in gene therapy, spotlighting patients, clinicians, and researchers who are redefining what's possible, including five-year insights from the HOPE-B study in hemophilia B.

This episode is a celebration of hope, trust, and progress—a reminder that hemophilia care isn't just about science, it's about people and the possibilities they create.

Presenting Sponsor:

Takeda, visit bleedingdisorders.com to learn more.

It's a Whole New World brought to you by @CSLBehring.

#bloodstreammedia #raredisease #bleedingdisorders #podcast #chronicdisease #genetherapy #healthcare #digitalhealth #biotech

Show Notes:

https://www.nejm.org/doi/full/10.1056/NEJMoa2514332

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On this episode of BloodStream, we look back at a year that reshaped the bleeding disorders community. Our Top Stories of 2025 segment explores the biggest shifts, from expanded treatment choices and growing complexity, to hard conversations about safety, trust, and the real-world limits of innovation. We examine gene therapy's reality-check moment, global progress through changes to the WHO Essential Medicines List, and why 2025 marked a turning point for women and girls with bleeding disorders.

Also featured, a powerful I'm Fine segment with Makenna Dietrich, who shares how "I'm fine" can hide anxiety and inaction, and why waiting has a cost. Her story is a reminder that speaking up and seeking support can bring clarity and change.

Together, these stories capture a year defined by honesty, courage, and a deeper understanding of what progress really asks of us.

Presenting Sponsor:

Takeda, visit bleedingdisorders.com to learn more.

I'm Fine is presented by @SanofiUS

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

Show Notes:

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In this special year-end episode of the Bloodstream Podcast, Patrick and guest host Mark Pangilinan conclude their powerful three-part Blood Brother series featuring the voices and stories of men living with bleeding disorders across the country. Together, they explore themes of complicated grief, advocacy, identity, fatherhood, discrimination, and the emotional realities that often go unspoken within the community. Listeners will also hear the latest installment of the Gene Therapy: A Whole New World segment, where Dr. Margaret Ragni and Dr. Richard Lemons break down new long-term data, future technologies, and where gene therapy may be heading next. It's an honest, heartfelt, and hopeful finale that celebrates vulnerability, connection, and the collective strength of the bleeding disorders community.

Presenting Sponsor:

Takeda, visit bleedingdisorders.com to learn more.

Gene Therapy Segment

It's a Whole New World brought to you by @CSLBehring.

#bloodstreammedia #raredisease #bleedingdisorders #podcast #chronicdisease #genetherapy #healthcare #digitalhealth #biotech

Show Notes:

To meet members of the community and to hear more about their hemophilia A journey, head

to www.hemophiliacommunity.com

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The BloodStream Podcast

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BloodStreamMedia.com

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