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

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

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 ...

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

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

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

European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

Sue Taylor with her son Will (Image: Muscular Dystrophy UK) Sam Taylor, who created the challenge, is joined by his wife Sue, 47, and daughter Beth, 17. They are taking part in support of his eldest ...

Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...

Genethon gets UK MHRA nod to initiate pivotal phase 3 trial of GNT0004, a low-dose microdystrophin gene therapy for Duchenne muscular dystrophy: Paris, France Wednesday, July 30, ...