The MDA's 75th anniversary conference highlights advancements in gene therapy, precision medicine, and patient-centered care for muscular dystrophy. Discussions focus on equitable access to ...
Aarhus, Denmark, 12 February 2026 – NMD Pharma A/S, a clinical-stage biotechnology company dedicated to developing novel therapies to restore skeletal muscle health, today announced that top-line data ...
Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, ...
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What is spinal muscular atrophy type 1 as Jesy Nelson's twins receive heartbreaking diagnosis
Over the weekend, Jesy Nelson took to social media to share the heartbreaking news that her 8-months-old twin daughters were diagnosed with a rare health condition. The former Little Mix singer ...
MDA 2026 keynote speaker John Crowley discussed with MD News Today the progress made in neuromuscular disease and the future ...
Breakthroughs across the neuromuscular field Right now, neuromuscular research is entering one of the most promising eras we have ever had. Dozens of new therapies are already changing lives, and many ...
MONDAY, Aug. 11, 2025 (HealthDay News) — Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the ...
ARTHEx Biotech, a clinical-stage biotechnology company advancing RNA-based therapeutics for neuromuscular disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast ...
HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1 -- 48-week trial will enroll approximately 150 ...
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