A Pfizer result is unsettling the gene therapy push for Duchenne muscular dystrophy: The boys didn't improve even though a ...

A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular ...

After months of uncertainty and controversy, the FDA has given approval to Sarepta's Duchenne muscular dystrophy drug – even though it hasn't yet shown any proof it can slow progress of the ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead ...

"My medicine is making me stronger," 7-year-old Hudson Sanford says after receiving a breakthrough gene therapy for Duchenne Muscular Dystrophy Cara Lynn Shultz is a writer-reporter at PEOPLE.

Duchenne muscular dystrophy (DMD) is a neuromuscular disease causing progressive skeletal muscle weakness and fatigue. The research team led by Associate Professor Hidetoshi Sakurai (Department of ...

A new investigation into Duchenne muscular dystrophy (DMD) pathogenesis suggests that at least part of the muscle degeneration observed in DMD patients may result from the reduced production of ...

CHMP upheld earlier opinions that Translarna is not of significant benefit to DMD due to nonsense mutations; European ...

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has once again snubbed Translarna ...

soared 51% Tuesday after the company reported positive interim data from a mid-stage trial of a treatment for the rare muscle disorder Duchenne muscular dystrophy. That put the stock on track for ...

Interim phase II data of Wave Life Sciences Ltd.’s oligonucleotide, WVE-N531, revealed “impressive” dystrophin expression, solid safety and the potential for once-monthly dosing for boys with Duchenne ...