Some types of muscular dystrophy cause irregular heartbeats. Gene therapy. Delandistrogene moxeparvovec (Elevidys) is a gene therapy approved to treat Duchenne MD. It's only prescribed to people ...

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD ... a "best-in-class therapy designed to suppress ...

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

The Next Chapter star Leah Messer opens up about protecting her daughter Ali amid her muscular dystrophy diagnosis, while also encouraging her to make her own decisions. The reality star also shares ...

Muscular Dystrophy (MD ... degeneration and medications like beta-blockers for cardiac complications. Physical therapy helps delay muscle contractures and maintain mobility, while occupational ...

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy ... measures of physical ability, although ...

The (MDA) has selected Lily Sander from Charlotte, North Carolina, as its 2025 MDA National Ambassador. Sander joins MDA ...

Duchenne muscular dystrophy (DMD ... the need for a wheelchair and effects of the disease on the heart. Physical therapy can ...

in children with the rare muscle-wasting disorder Duchenne muscular dystrophy (DMD). The phase 3 EMBARK trial has been evaluating the gene therapy in ambulatory boys aged four to seven years with a ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years in patients with Duchenne muscular dystrophy (DMD).

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

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