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 ...

Mutations that lead to muscle atrophy can be repaired with the gene editor CRISPR-Cas9. A team led by ECRC researcher Helena Escobar has now introduced the tool into human muscle stem cells for the ...

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 ...

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

PHILADELPHIA, May 14, 2026 /PRNewswire/ -- Gemma Biotherapeutics ("GEMMABio"), a clinical‑stage, global, genetic medicines company, today announced the presentation of preclinical data supporting ...

As someone who has lived with spinal muscular atrophy for all 30 years of my life, I was perplexed and disappointed that the recent approval of Novartis’ gene therapy Zolgensma was immediately ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic ...

Spinal muscular atrophy is a progressive neurodegenerative disease that affects the central nervous system, peripheral nervous system and voluntary muscle movement, according to the Muscular Dystrophy ...

At her eight-month pregnancy check-up, Lizmara Figueroa met with a new doctor. As he walked out the door, he made a parting comment. “He says, ‘Oh, just so you know, you’re a carrier for (spinal ...

National September campaign celebrates awareness across the neuromuscular disease community through storytelling, social media, events, and fundraising. September is National Muscular Dystrophy ...