7 August 2020 - The U.S. Food and Drug Administration today approved Evrysdi (risdiplam) to treat patients two months of age and older with spinal muscular atrophy, a rare and often fatal genetic disease affecting muscle strength and movement. 

This is the second drug and the first oral drug approved to treat this disease.

The efficacy of Evrysdi for the treatment of patients with infantile-onset and later-onset spinal muscular atrophy was evaluated in two clinical studies.

Read FDA press release