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 ...

In many genetic diseases, a small error in the DNA code can have devastating effects. These errors, called nonsense mutations ...

John Davidson and his 15-year-old son Jesse captured the hearts of Canadians when John pushed Jesse across Ontario in his ...

Maha Radhakrishnan, MD Maha Radhakrishnan, MD BOSTON, June 03, 2025 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: ...

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

In cohort 6, subjects aged two years old at the time of treatment showed a mean expression of 93.87% of normal dystrophin ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec. AAN Evidence in Focus articles highlight the strength of the current ...

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

Canadians are walking, running, and rolling in support of the 30th annual Walk to Defeat Duchenne, the country's only national ...

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

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 ...