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

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

Nisa Investment Advisors LLC grew its position in shares of Sarepta Therapeutics, Inc. (NASDAQ:SRPT – Free Report) by 23.1% ...

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

Sarepta Therapeutics (NASDAQ:SRPT – Get Free Report)‘s stock had its “buy” rating reiterated by research analysts at Needham ...

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

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 ...

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

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

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

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