Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early diagnosis, multidisciplinary care, and emerging treatments like gene therapy ...

The Next Chapter star Leah Messer opens up about protecting her daughter Ali amid her muscular dystrophy diagnosis, while ...

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 gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

But if this therapy works, it will heal the muscle." For nearly 20 years, Spuler and her collaborators have been working to understand dysferlin, its role in muscular dystrophy and ways to cure ...

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 ...

But if this therapy works, it will heal the muscle.” For nearly 20 years, Spuler and her collaborators have been working to understand dysferlin, its role in muscular dystrophy and ways to cure ...

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

But if this therapy works, it will heal the muscle.” For nearly 20 years, Spuler and her collaborators have been working to understand dysferlin, its role in muscular dystrophy and ways to cure these ...

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

What Is Duchenne Muscular Dystrophy? Muscular dystrophies are ... The doctor will give your child a physical exam, and they may do some tests to rule out other conditions that can cause muscle ...