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

Sarepta Therapeutics’ gene therapy to treat limb girdle muscular dystrophy type 2E/R4 gets US FDA platform technology designation: Cambridge, Massachusetts Friday, June 6, 2025, ...

Regenxbio (RGNX) stock in focus as the company shares promising trial data for its RGX-202 gene therapy for Duchenne muscular ...

RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that ...

Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...

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

Repurposing existing therapies may help slow muscle weakness in patients with Duchenne muscular dystrophy, a new study ...

Satellos Bioscience's experimental small-molecule drug for Duchenne muscular dystrophy is showing positive results as a stand-alone treatment, but Phil Lambert, Ph.D., the company's chief scientific ...

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

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a ...

The campaign to commemorate the work of the Muscular Dystrophy Association was created in partnership with agency Yes&.