The inflammation associated with DMD was also improved by the treatment as assessed by a reduction ... This trial is clearly limited by its small size and lack of clinical outcomes.

The company is evaluating whether the drug, which targets cardiac complications, might be used treat patients with other ...

Market analysts said that investors' decisions might have been a slight overreaction, because unless Sarepta's gene therapy fully compensates for the lack of dystrophin production in DMD patients ...

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

While DMD is currently incurable, hope lies in emerging treatments like ELEVIDYS (Delandistrogene Moxeparvovec Rokl), a gene therapy designed to help the muscles produce dystrophin. However ...

Currently, the DMD treatment landscape includes Sarepta’s Elevidys (delandistrogene moxeparvovec), the first FDA-approved gene therapy for DMD, which delivers a micro-dystrophin gene via an ...

Sarepta Therapeutics (NASDAQ:SRPT), a biotechnology company specializing in precision genetic medicines for rare diseases with a market capitalization of $11.2 billion, stands at a critical juncture ...

as a gene therapy for treating Duchenne muscular dystrophy (DMD). Roche entered into a worldwide partnership with Sarepta Therapeutics in 2019 to commercialise the gene therapy outside of the US.

The announcement was made at Arab Health 2025 where Sidra Medicine will highlight its international patient service programmes ...