Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, ...
New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of individuals with Types 2 or 3 spinal muscular atrophy (SMA) Late-breaking ...
We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Patients treated with onasemnogene abeparvovec recorded ...
With a tank of zebrafish, Queensland researcher Jean Giacomotto was able to test a never-before seen genetic variant ...
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 ...
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic ...
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 ...
Manistee News Advocate on MSN
AMISH KITCHEN: Daughter Verena discusses her disability
"Muscular dystrophy can take many things from me, but it will not take away my happiness, nor will it take away my faith." ...
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