by Peter Ciszewski | Jul 17, 2019
Odylia Therapeutics is a non-profit corporation working to help develop gene therapies for rare eye disease. Recently, we talked to Harrison Brown, PhD, BBA, the company’s Chief Science Officer about advances in gene therapy. As Dr. Brown explains in this...
by Peter Ciszewski | Jul 16, 2019
Mark Pykett, PhD, Chief Scientific Officer at PTC Therapeutics, discusses three of his company’s clinical programs, including Spinal Muscular Atrophy, Familial Dysautonomia (FD) and AADC Deficiency. Spinal Muscular Atrophy is a genetic disease caused by...
by Peter Ciszewski | Jul 15, 2019
Lynn O’Connor Vos, President and Chief Executive Officer at the Muscular Dystrophy Association, discusses her organization and it’s approach to funding research to accelerate treatments and cures across the full spectrum of neuromuscular...
by Peter Ciszewski | Jul 15, 2019
Lynn O’Connor Vos, President and Chief Executive Officer of the Muscular Dystrophy Association (MDA) provides an overview of her association. The MDA is committed to transforming the lives of people affected by muscular dystrophy, ALS, and related...
by Peter Ciszewski | Jul 12, 2019
Heather A. Lau, MD, Assistant Professor, Department of Neurology; Associate Director, Division of Neurogenetics; Director, Lysosomal Storage Disease Program at NYU Langone Health, discusses some of the typical patient features across all subtypes of...
