CINCINNATI (WKRC) - The annual "Cure SMA Walk-n-Roll" event kicked off Saturday morning at Xavier University, drawing ...

From a missing sign turning up across the Irish sea, to cancelled festivals and teenagers locked in a play park - here are ...

Months after causing safety concerns that led the FDA to hit a related trial with a clinical hold, a phase 2 trial of ...

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

Families began receiving calls from doctors last week to arrange treatments for new Givinostat drug after a campaign by the ...

A rare and fatal form of muscular dystrophy has long posed a devastating diagnosis for children, primarily affecting boys.

The "Duchenne Muscular Dystrophy - Global Clinical Trials Review, 2025" report provides a comprehensive overview of the global clinical trials landscape for Duchenne Muscular Dystrophy. It includes ...

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a ...

Drug developer PepGen said on Wednesday it will discontinue development of its experimental therapy for Duchenne muscular dystrophy (DMD) after it failed to boost production of a protein in a ...

Living with a rare form of muscular dystrophy, and being honoured with having the youngest-ever seat on the board of trustees ...

PepGen ends Duchenne drug program after weak Phase 2 results, pivots to DM1 treatment showing early signs of splicing ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following ...