Dystrophin's isoforms are thought to contribute variably to neuropsychiatric disorders, behavioral disorders, and ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...

Intellia’s stock fell 23% after one patient in its Phase III MAGNITUDE trial developed severe liver toxicity that later ...

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

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

CureDuchenne, a global nonprofit based in Newport Beach committed to finding and funding a cure for Duchenne muscular ...

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

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 ...

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

A 46-year-old woman with type 1 myotonic dystrophy (DM1) presented to the emergency department with dyspnea and palpitations.

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