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 ...
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 ...
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 ...
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) ...
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 ...
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Jesy Nelson Reveals Twins' Rare Diagnosis—What Parents Need To Know About Spinal Muscular Atrophy
British singer Jesy Nelson recently took to Instagram to share a devastating update about her 8-month-old twin daughters.
Jesy Nelson revealed her twin daughters have been diagnosed with spinal muscular atrophy (SMA) in an emotional video while ...
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 ...
Health Secretary was responding to news shared by the former Little Mix star that her twin daughters have been diagnosed with ...
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