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 ...

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 ...

Roche’s autoimmune drug Enspryng is making solid progress as a treatment for neuromyelitis optica spectrum disorder (NMOSD).

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 ...

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 ...

When Ingram became Sarepta Therapeutics’ CEO in 2017, he didn’t have a connection to muscular dystrophy, but he has developed ...

Next week, researchers, clinicians, industry leaders, and families will gather at the 2026 MDA Clinical & Scientific Conference, hosted by the Muscular Dystrophy Association (MDA), to discuss the ...

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 ...

Muscular Dystrophies are a group of genetic conditions characterized by muscle weaknesses. They are genetic conditions and have different sub-categories depending on which area of muscle mass is ...

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 ...

"High clinical suspicion is important for this population of patients. Since muscular dystrophy is progressive in nature, monitoring disease progression in these patients is critical for timing ...