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

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

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

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

During this season of hope, columnist Robin Stemple shares his wishes for both the disability community and himself.

Experimental therapy deramiocel outperformed a placebo at improving measures of arm and heart health in people with DMD in a ...

Dyne Therapeutics, citing trial data, said it will ask the FDA to approve DYNE-251, its therapy for DMD amenable to exon 51 skipping.

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 ...

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 ...

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 ...

Muscular dystrophy refers to a group of genetic diseases that cause progressive muscle weakness and loss. There are more than 30 types of muscular dystrophy, all with different causes and symptoms.