“Mutations in the dystrophin gene lead to progressive muscle fiber degeneration and weakness.” And as Sanford tells PEOPLE, “When you Google it, it's really devastating. It's a horrible disease.

This reduction may lead to impaired regulation of the vasoconstrictor response and eventual muscle damage. Mutations in the gene encoding dystrophin cause X-linked Duchenne muscular dystrophy (DMD ...

Specific mutations in the DMD gene influence how long patients retain the ability to walk, even when treated with ...

The accelerated approval of Exondys 51 is based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some Exondys 51-treated patients. In this case the FDA said Sarepta ...

In this condition, patients' bodies don't make enough for the dystrophin protein necessary to keep muscles intact. Sarepta ...

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

Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead to malfunction or absence of dystrophin. This protein stabilizes the ...

The $514,000 DOD award will be used to study the efficacy of gene editors in the treatment of Duchenne Muscular Dystrophy.

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 ...

The 9% adjusted figure reflects the ability of the drug to penetrate into muscle tissue at high concentrations, said Wave in a letter to the DMD patient community, which reads: "Dystrophin was ...

I started calling myself an artist in 2019. And that is what I do, I make art. I make really good art. I even have a degree ...

SAT-3247 presents a novel mechanism of action to restore impaired muscle regeneration caused by the absence of functional dystrophin. About Duchenne Muscular Dystrophy Duchenne muscular dystrophy ...