Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to ...

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...

A mouse model study of Precision BioSciences’ gene-editing therapy PBGENE-DMD found it produced long-term functional ...

The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...

European Medicines Agency recognizes potential of lead compound MP1032 in addressing a high unmet medical need in Duchenne Muscular Dystrophy (DMD)Designation is paving the way for safer long-term ...

On Thursday 28 August, Sam Taylor, 46, his wife Sue, 47, and daughter Beth, 17, from Crooklands in Cumbria will set off to ...

Genethon is determined to bring GNT0004 to market for young patients and their families who are waiting for a therapeutic ...

FDA probes Elevidys after an 8-year-old's death in Brazil; Sarepta and Roche say the death was unrelated to gene therapy ...

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...

The FDA greenlit multiple new drugs this month and issued some notable label expansions, including for Eli Lilly’s Kisunla.