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

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

Duchenne muscular dystrophy (DMD) treatment ifetroban led to improvements in measures of heart function in a Phase 2 clinical ...

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

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

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

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from ...

The gene therapy improved motor functions in children with Duchenne muscular dystrophy two years after treatment ...

Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX) shares began Tuesday sharply higher. Cumberland, a specialty pharmaceutical company with development efforts focused on new products for rare diseases, ...

Roche announces positive results from EMBARK phase III study of Elevidys in ambulatory individuals with DMD: Basel Tuesday, January 28, 2025, 11:00 Hrs [IST] Roche announced posit ...

Entrada Therapeutics plans to initiate ELEVATE-44-201 trial for ENTR-601-44 in Duchenne muscular dystrophy by Q2 2025 ... including exon skipping and dystrophin production in approximately ...