While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...

Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early ...

In year two of the trial, Elevidys demonstrated statistically significant and clinically improvements across three key ...

H.C. Wainwright adjusted its financial outlook for PepGen Inc. (NASDAQ:PEPG), reducing the price target to $16.00 from the ...

A Vancouver family is sending their son on one final journey into the stars, where his ashes will eventually transform into a ...

Roche and Sarepta Therapeutics have shared positive top-line results from a late-stage study of Elevidys (delandistrogene ...

Prophylactic cardiac treatment may prolong survival in Duchenne muscular dystrophy (DMD), but only one-quarter of individuals ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

PepGen (PEPG) “provided recent updates on its CONNECT clinical program investigating PGN-EDO51 in Duchenne muscular dystrophy for patients ...

Initiation of Phase 1b study using SGT-212 for the treatment of patients with Friedreich's Ataxia expected in 2H25. Click ...