Positive Data from FIREFISH Study of Evrysdi™ (risdiplam) in Infants with Type 1 Spinal Muscular Atrophy Published in The New England Journal of Medicine Results found that patients treated with Evrysdi at 12 months demonstrated significant improvements in survival and developmental milestones The U.S. Food and Drug Administration approved Evrysdi in August 2020 as the first and only at home SMA treatment with proven efficacy in adults, children and infants 2 months and older
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SOUTH PLAINFIELD, N.J., Feb. 24, 2021 /PRNewswire/ PTC Therapeutics, Inc. (NASDAQ: PTCT), today announced the publication of results from the dose finding Part 1 of the pivotal FIREFISH study evaluating Evrysdi™ (risdiplam) in infants with symptomatic type 1 spinal muscular atrophy (SMA) in The New England Journal of Medicine (NEJM)