Dystrophin is absent in patients with DMD, so when it’s reintroduced via gene therapy, the immune system may perceive it as a foreign protein, an antigen, triggering a response that eliminates ...

DMD is a progressive, inherited muscle-wasting disease primarily affecting boys, caused by a lack of the protein dystrophin ... estimates that should DYNE-252 make it to market, it is predicted ...

RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin ... to produce the protein known as dystrophin, it is this protein that ...

- Potential first-in-class gene editing approach designed for dystrophin gene correction leading the body to produce a functional dystrophin protein applicable for majority of DMD patients (up to ...

Biopsy data from eight of the boys showed a 6.4% mean muscle content-adjusted dystrophin ... benefit seen one year after taking Sarepta’s DMD gene therapy Elevidys. The 48-week data for Wave ...

Micro-dystrophin expression was ... moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that benefits in functional outcomes, gene expression, and muscle imaging persist 2 years ...

One patient with Duchenne muscular dystrophy (DMD ... the administered gene produces microdystrophin, a shortened version of the normal dystrophin protein that’s missing in Duchenne patients.