We approved the first therapy to directly target muscle weakness linked to muscle loss in patients with the rare disease of spinal muscular atrophy (SMA). Designed to work alongside existing SMA treatments, it helps patients age 2 and older improve their strength and function. This is a meaningful milestone for people living with spinal muscular atrophy, and another step forward in FDA’s commitment to advancing treatments for people with rare diseases. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-therapy-target-muscle-loss-spinal-muscular-atrophy