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

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

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

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

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

Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver ...

BASEL, Switzerland I July 25, 2025 I Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the ...

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

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

CHMP issues negative opinion on conditional marketing authorization for Roche’s gene therapy, Elevidys for Duchenne muscular dystrophy: Basel Monday, July 28, 2025, 15:00 Hrs [I ...

The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...

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, ...