The FDA rejected a Duchenne muscular dystrophy cell therapy from Capricor Therapeutics, as a larger study of the treatment ...

Monitoring Duchenne muscular dystrophy helps ensure timely treatment. ... Your child should have a dual-energy X-ray absorptiometry (DEXA) test to measure bone density every 2-3 years.

“Duchenne muscular dystrophy (DMD) ... In the STRIDE Registry study, ataluren was associated with preserved hand-to-mouth function by 3.4 years (P = .0046).

The company said it was “surprised” by the decision, which followed the ouster of cell and gene therapy officials from the ...

Like Duchenne muscular dystrophy, Becker muscular dystrophy typically affects only males (1 in 30,000) and causes heart problems. Disease severity varies. Those with Becker can usually walk into ...

Dysfunctional dystrophin, the muscle protein affected in Duchenne, leads to an important choice for patients about how to ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

New consensus guidelines outline best practices for orthopedic management in Duchenne muscular dystrophy, focusing on fracture prevention and surgical care across pediatric and adult populations.

A new gene therapy for Duchenne muscular dystrophy (DMD) has shown promise in not only slowing the progression of the disease but potentially even reversing the muscle damage.

Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus. Neurology , 2025; 104 (11) DOI: 10.1212/WNL.0000000000213604 Cite This Page : ...

Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular dystrophy. Accessibility statement Skip to main content.