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News From the Food and Drug Administration
September 15, 2020

Oral Drug Approved for Spinal Muscular Atrophy

JAMA. 2020;324(11):1026. doi:10.1001/jama.2020.16783

The first oral therapy for adults and children aged 2 months or older who have spinal muscular atrophy (SMA) has received FDA approval.

A fatal genetic disease that leads to severe muscle weakness, SMA affects about 1 in 8000 to 10 000 people worldwide. Infants with SMA can have difficulty sitting upright, controlling their head movements, swallowing, and breathing. Children with the most common form, type 1, typically do not survive past early childhood.