PTC Therapeutics (PTCT) Positive Data from FIREFISH Study of Evrysdi in Infants with Type 1 SMA Published in NEJM

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Go back to PTC Therapeutics (PTCT) Positive Data from FIREFISH Study of Evrysdi in Infants with Type 1 SMA Published in NEJM

Positive Data from FIREFISH Study of Evrysdi™ (risdiplam) in Infants with Type 1 Spinal Muscular Atrophy Published in The New England Journal of Medicine

February 24, 2021 5:08 PM EST

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). The Part 1 data showed that patients treated with Evrysdi at 12 months demonstrated significant improvement in survival and developmental milestones, including the ability to survive without permanent ventilation, and the ability to sit without support for at least five seconds. Data from the... More