Jennifer McNary, rare disease advocate, looks back at the Sarepta AdComm for Exondys 51 in 2016. She also discusses the role of the patient advocate.
Len Walt, Vice President, Head of Medical Affairs, SOBI in North America, discusses his company’s activities in Hemophagocytic lymphohistiocytosis (HLH), a rare condition in which the body makes too many activated immune cells (macrophages and lymphocytes).
Jennifer McNary is a consultant, speaker, and rare disease advocate. She is also a mother of two boys with Duchenne muscular dystrophy (DMD). In this video, Jennifer discusses her two boys, Max and Austin, and their journey with DMD as well as the some of the emerging therapies that other …
Jennifer McNary, Rare Disease Advocate, explains why there is so much interest in Duchenne muscular dystrophy and some of the future considerations for this rare diease.
Jodie Gillon Global Medical Lead, Patient Engagement Rare Diseases at Pfizer discusses Pfizer’s role in patient advocacy.
Greg Duncan, President and Chief Executive Officer of Celtaxsys, discusses working with advoacy groups such as the Cystic Fibrosis Foundation, on their clinical trials.