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Rare Disease Film Festival: Pyruvate Kinase Deficiency Advocacy

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Callie Mertel, Conference Producer for Informa Connect, sits down for a chat with Tamara Schryver (middle) and  Alejandra Watson (right), leaders of two pyruvate kinase (PK) deficiency advocacy groups. Learn how this rare blood disorder community grew from a private Facebook group to an active, vocal community that advocates for itself. The advocacy leaders share their experiences of meeting with the FDA in a patient-focused drug development seminar, the formation of a multi-stakeholder advocacy advisory council (AAC), and the launch of two independent U.S.-based advocacy groups. This film was sponsored by Agios Pharmaceuticals.

 

 

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