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 ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced its participation at the 2026 Muscular Dystrophy Association (MDA) Clinical and ...

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 ...

Muscular dystrophies are a group of more than 30 genetic disorders characterized by progressive muscle weakness and wasting. These disorders are typically associated with defects in muscle ...

The agency acted after reviewing mouse data submitted in 2024, making the timing of the new pause “confusing,” one analyst wrote.

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 ...

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 ...

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

Why hello everyone! And welcome to a new column I like to call A Disabled Woman’s Guide. OK, it’s a working title, but you get the idea!