The Muscular Dystrophy Association calls FDA approval of Novartis' Itvisma (onasemnogene abeparvovec-brve) a major step forward for the spinal muscular atrophy community. The Muscular Dystrophy ...

FDA Approves High Dose Regimen of SPINRAZA® for Spinal Muscular Atrophy, Reflecting Progress Made Possible Through Decades of MDA Supported Research The Muscular Dystrophy Association celebrates FDA ...

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, Sept. 11, 2026 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) marks another milestone for the neuromuscular community with the U.S. Food and Drug Administration (FDA) approval ...

Reflex sympathetic dystrophy is a painful, disabling disorder of unknown pathophysiological origin that usually commences after trauma to or surgery on a limb. In chronic cases, the syndrome leads to ...

Long-term data from DEVOTE/ONWARD studies show benefits of high dose nusinersen in people living with spinal muscular atrophy (SMA) New Phase 1b data further illustrate potential of salanersen in SMA, ...

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 recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene transfer ...

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FDA Approves Isembyld (apitegromab-mstn), the First and Only Muscle-Targeted Therapy for Spinal Muscular Atrophy, Marking a New Milestone in SMA Treatment The Muscular Dystrophy Association marks a ...