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 ...

The FDA has granted rare pediatric disease designation to (Z)-Endoxifen as a potential treatment for Duchenne muscular dystrophy.

As 2025 draws to a close, columnist Shalom Lim looks back at the highs and lows he's experienced this year while living with ...

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 ...

Muscular dystrophy (MD) refers to a group of inherited muscle disorders caused by mutations in genes that generate proteins that play an essential role in muscle structure and function. The disease ...

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

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.

As part of an effort to heighten awareness of its work in research, treatment and care — as well as to raise funds — the Muscular Dystrophy Association (MDA) has proclaimed September MDA National ...

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 ...

Capricor Therapeutics will resubmit its application for U.S. Food and Drug Administration (FDA) approval of its cell therapy deramiocel to treat Duchenne muscular dystrophy (DMD)-related heart disease ...