Rare Disease Film Festival: Pyruvate Kinase Deficiency Advocacy

Hear from the leaders of two pyruvate kinase (PK) deficiency advocacy groups, and 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.

Видео Rare Disease Film Festival: Pyruvate Kinase Deficiency Advocacy канала Informa Connect Life Sciences