Expanding Possibilities in the Treatment of Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an inherited, degenerative neuromuscular disease that can result in severe muscle weakness and loss of function over time. Disease severity and rate of functional decline vary, ranging from hypotonia, failure to reach motor milestones, and potentially fatal respiratory complications in infantile-onset SMA, to loss of ambulation and slow but progressive motor function decline in children and adults with later-onset disease. In this session, participants will be introduced to efficacy and safety data from an approved SMA treatment option.