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

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 ...

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 ...

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 ...

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.

For parents and caregivers, a new diagnosis of Duchenne muscular dystrophy (DMD) can feel overwhelming. There’s no road map to managing the physical and mental changes in your child’s health, ...