Duchenne muscular dystrophy (DMD) causes dysfunction of muscle cells that help move blood through the circulatory system, a ...

Life is so much easier with a sense of humor, writes columnist Patrick Moeschen, because with LGMD, there’s no shortage of ...

Dyne Therapeutics said it plans to ask the U.S. Food and Drug Administration (FDA) to approve DYNE-251, its exon-skipping therapy for people with Duchenne muscular dystrophy (DMD) amenable to exon 51 ...

Advocates are celebrating a recent U.S. federal decision by HHS recommending that all newborns be screened for Duchenne muscular dystrophy.

A young man with Duchenne muscular dystrophy (DMD) who received the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) has died due to acute liver failure. In a statement, the ...

Doctors can use several tests to diagnose muscular dystrophy (MD). A correct diagnosis gives patients an understanding of how their disease is likely to progress. It also helps doctors do a better job ...

Histone deacetylase inhibitors are a treatment approach designed to work by blocking an enzyme that turns off gene activity. This may ultimately help by slowing muscle loss and disease progression.

It’s been nearly two years since Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for Duchenne muscular dystrophy (DMD) developed by Sarepta Therapeutics, was approved by the U.S. Food and ...

People with Duchenne muscular dystrophy (DMD) require some level of caregiving throughout their whole lives, particularly as the disease progresses and patients become less mobile and more reliant on ...

For teenagers or young adults with Duchenne muscular dystrophy (DMD), learning life skills can help you gain more control over your life while managing this progressive condition. For parents, helping ...

After three years of treatment with Capricor Therapeutics’ experimental cell therapy CAP-1002, people with Duchenne muscular dystrophy (DMD) continue to show benefits in arm and heart function, new ...

Sarepta Therapeutics plans to begin a clinical trial to test the antibody-cleaving therapy imlifidase as a pre-treatment for SRP-9001 (delandistrogene moxeparvovec) — its experimental gene therapy for ...