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, ...
Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced presentations on SGT-003, its ...
The MDA meeting is a key opportunity to highlight the expanding real‑world evidence shaping care for the Duchenne community,” said Will Andrews, MD, Chief Medical Officer of Catalyst. “Through our ...
Seven-year-old William, a Southern Oregon boy living with Duchenne muscular dystrophy, is going to get a never-before-tried surgery designed specifically for his genetic mutation. NBC5’s medical ...
Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...
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First successes in the development of a gene therapy for incurable LAMA2-related muscular dystrophy
Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...
A RECOMMENDATION for the HSE not to cover the cost of a drug to help children with a rare muscular disease has been blasted by families. Last year, The Irish Sun highlighted the plight of ...
Roche’s autoimmune drug Enspryng is making solid progress as a treatment for neuromyelitis optica spectrum disorder (NMOSD).
Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced its participation at the 2026 Muscular Dystrophy Association (MDA) Clinical and ...
The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...
While it’s established that Duchenne muscular dystrophy (DMD) causes a loss of muscle mass and strength, few studies have examined the gastrointestinal issues tied to DMD. Initial fractures are the ...
This Monday 9 March, the nation is invited to celebrate National Meatball Day with purpose, by turning its love of meatballs ...
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