Some of the market’s biggest winners start off as the smallest players. While trillion-dollar tech giants like the ...

There is an unmet need for therapies targeting Duchenne muscular dystrophy (DMD)–related heart disease, and phase 2 results ...

A seven-year-old boy from Dublin is on his way to California to undergo testing after being diagnosed with a rare condition last year.

Cumberland Pharmaceuticals Inc. (NASDAQ:CPIX) released topline results from its Phase 2 FIGHT DMD trial. The study evaluated ...

Entrada plans to initiate clinical testing of ENTR-601-44 in Duchenne MD patients amenable to exon 44 skipping later this year.

"Connections with Evan Dawson" brings you special rebroadcasts on 1/30/25. First, AI expert Landon Gray on the future of ...

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

The positive results from the second part of the EMBARK trial contrast with findings from one-year post treatment, which did ...

Sarepta (SRPT) announced topline results from Part 2 of EMBARK, a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study ...

(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of ...

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