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 ...

Entrada Therapeutics has secured authorisation from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) to ...

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

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 ...

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

Despite the obstacles, Riley Herrera had a life filled with family, sports, travel, faith and hope before his death on Nov.

Sarepta's EMBARK study confirms Elevidys offers sustained Duchenne treatment benefits, including motor function improvement ...

Top-line Phase 3 trial data show ambulatory boys with Duchenne continued with motor improvements two years after a single ...

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

Equities researchers at Zacks Research lifted their Q4 2024 earnings estimates for Sarepta Therapeutics in a research note issued to investors on Monday, February 3rd. Zacks Research analyst S.

Roche has reported positive results from the second year of the EMBARK Phase III trial of Elevidys (delandistrogene moxeparvovec), the first approv ...