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 ...

Health Secretary was responding to news shared by the former Little Mix star that her twin daughters have been diagnosed with ...

Scholar Rock Holding Corp. (NASDAQ:SRRK) said on Sunday the company will present new efficacy and safety data from the Phase 3 pivotal SAPPHIRE trial at the 2025 Muscular Dystrophy Association (MDA) ...

Highlights from the 2025 Muscular Dystrophy Association (MDA) Conference include clinical updates, expert insights, and breaking trial findings.

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 ...

Jesy Nelson revealed her twin daughters have been diagnosed with spinal muscular atrophy (SMA) in an emotional video while ...

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 ...

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 ...

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 ...