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 ...

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 ...

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 ...

Columnist Betty Vertin is planning a holiday season this year that will let her sons with DMD better enjoy their time with family and friends.

What is Elevidys for Duchenne muscular dystrophy? Elevidys (delandistrogene moxeparvovec-rokl), previously known as SRP-9001, is a one-time gene therapy approved in the U.S. to treat patients with ...

Data from two clinical trials confirm that Agamree (vamorolone), an approved anti-inflammatory treatment for people with Duchenne muscular dystrophy (DMD), blocks the action of a receptor involved in ...