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FDA approves Evrysdi (risdiplam) for treatment of Spinal Muscular Atrophy (SMA) in adults and children 2 months and older

The SMA Foundation’s partners, Roche and PTC Therapeutics, announced today that Evrysdi™ (risdiplam) was approved by the U.S. Food and Drug Administration (FDA) for the treatment of Spinal Muscular Atrophy (SMA) in adults and children two months and older. Evrysdi showed clinically meaningful improvements in motor function across two clinical trials covering a broad range of ages and levels of disease severity. Evrysdi also demonstrated a favorable safety profile. A liquid medicine, Evrysdi is the first and only medicine for SMA that can be taken at home.

For more information on the approval, please see press releases from Genentech/Roche and PTC Therapeutics.

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