The MarketWatch News Department was not involved in the creation of this content. New York, Nov. 24, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today called the U.S. Food and ...
Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...
Newly approved SMA therapies show promise but face global accessibility challenges, widening healthcare disparities due to high costs and limited availability. Newborn screening for SMA is widespread ...
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 [email protected]. Patients treated with onasemnogene abeparvovec recorded ...
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 ...
Jesy Nelson reveals her twins may never walk, but what is spinal muscular atrophy? - Jesy Nelson reveals her twin daughters have been diagnosed with SMA, a severe muscular disorder ...
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 ...
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