Continued Improvements in Motor Milestones in Infants with Type 1 Spinal Muscular Atrophy After Two Years of Treatment with Evrysdi™
- Evrysdi increased survival and reduced need for permanent ventilation -
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SOUTH PLAINFIELD, N.J., April 15, 2021 /PRNewswire/ PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that results from Part 2 of the pivotal FIREFISH trial demonstrated that infants with type 1 spinal muscular atrophy (SMA) treated with Evrysdi™ (risdiplam) obtained increases in survival and sustained improvements in achieving key motor milestones, including head control, sitting, rolling over, and further developing towards acquiring the ability to stand, and walk. Data were presented at the 2021 American Academy of Neurology (AAN) Virtual Annual Meeting.
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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)