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

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

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

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 ...

Columnist Patrick Moeschen argues that we all should focus more on finding the right words to accurately describe living with ...

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 ...

Exon skipping is a treatment approach for people whose Duchenne muscular dystrophy is due to certain mutations in its causative gene. Exon skipping works like a molecular patch, so that the DMD gene ...

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.