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 …
Greg Duncan, President and Chief Executive Officer of Celtaxsys, discusses working with advoacy groups such as the Cystic Fibrosis Foundation, on their clinical trials.
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses the important role of advocacy groups in rare diseases.
Cory MacDonald, Operations Manager, Choroideremia Research Foundation, discusses choroideremia, his advocacy organization, and the various clinical trials for this rare eye disorder.