feb 11, 2019: 11 a.m. - middag
After a long era without progress, recent years have seen regulatory approvals of treatments for Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA), among other neuromuscular disorders. In particular, SMA is the most common genetic disorder leading to infant death in the world, and now has a first therapy that can slow disease progression, giving patients and families more time together. This session will examine the exciting pipeline of additional neuromuscular disease therapies, and what the underlying science is teaching across disease indications, amid a complicated reimbursement context where the shortage of effective treatments bears significant healthcare costs over time.
Moderator: Lynn O'Connor Vos, President and CEO, Muscular Dystrophy Association (MDA)