On Aug. 7, 2020, the U.S. Food and Drug Administration (FDA) approved Genentech’s Evrysdi (risdiplam) to treat patients two months of age and older with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement. This was the second drug and the first oral drug approved to treat this disease.

SMA is a hereditary disease that causes weakness and muscle wasting because patients lose lower motor neurons (nerve cells) that control movement. Evrysdi contains a survival of motor neuron 2-directed RNA splicing modifier.