July 2021

Data for Genentech’s Evrysdi Published in New England Journal of Medicine Shows Significant Improvement in Survival and Motor Milestones in Babies with Type 1 SMA

Leave a Comment / Clinical Trials, Front Page News, Industry Updates, Research /

Data from Genentech, a member of the Roche Group, recently announced that the New England Journal of Medicine (NEJM) has published data from FIREFISH Part 2, a pivotal global study evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies aged 1-7 months old with symptomatic Type 1 spinal muscular atrophy (SMA). The study met its primary endpoint […]

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Cure SMA Convenes Working Group to Discuss Biomarkers for SMA

Leave a Comment / Front Page News, Research /

The advent of three approved SMN-upregulating therapeutics for spinal muscular atrophy has dramatically changed the natural history of the disease. There are, however, many uncertainties regarding timing of treatment initiation, response to treatment, and long-term outcomes.  The identification of biomarkers, medical indicators that can inform us as to disease status, treatment response, and/or provide information

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