We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Patients treated with onasemnogene abeparvovec recorded ...
Bipartisan Congressional Action Advances Critical Programs for Rare Neuromuscular Disease Research and Care Muscular ...
With a tank of zebrafish, Queensland researcher Jean Giacomotto was able to test a never-before seen genetic variant affecting two babies.
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic ...
Panelists discuss how exciting ongoing research efforts are expanding similar gene therapy technologies to other muscular dystrophies like FSHD and myotonic dystrophy, using strategies to knock down ...
New York, Nov. 24, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today called the U.S. Food and Drug Administration’s approval of Itvisma (onasemnogene abeparvovec-brve), developed ...
A trial of Biogen’s spinal muscular atrophy (SMA) therapy Spinraza (nusinersen) suggests that a higher dose provides benefits to a broad range of patients—including those who have and have not been ...
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