Over 95% of babies now diagnosed with SMA are treated with Zolgensma gene therapy, per Daniel Grant, vice president for ...

New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing ...

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Dec. 16, 2024 — A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an ...

The Cambridge-based biotech's experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, ...

Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the ...

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death ...

For millions of people, losing muscle isn't just about weakness; it's about losing independence. Whether caused by Duchenne muscular dystrophy, aging or other degenerative conditions, muscle loss can ...

WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients ...