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Spinal Muscular Atrophy News

FDA Approves Label Extension for Evrysdi for Infants with Spinal Muscular Atrophy Under 2 Months Old

SOUTH PLAINFIELD, N.J., May 31, 2022 /PRNewswire/ – PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has approved a label extension for Evrysdi...

FDA Approves Evrysdi (risdiplam) for Treatment of Spinal Muscular Atrophy (SMA) in Adults and Children 2 Months and Older

South San Francisco, CA – August 7, 2020 – Genentech, a member of the Roche Group, announced today that the U.S. Food and Drug Administration (FDA) has approved Evrysdi (risdiplam) for treatment of...

FDA Approves Zolgensma (onasemnogene abeparvovec-xioi) Gene Therapy to Treat Pediatric Patients with Spinal Muscular Atrophy

May 24, 2019 – The U.S. Food and Drug Administration today approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with...

FDA Approves Spinraza (nusinersen) for Spinal Muscular Atrophy

December 23, 2016 – The U.S. Food and Drug Administration today approved Spinraza (nusinersen), the first drug approved to treat children and adults with spinal muscular atrophy (SMA), a rare and...

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