In the Phase 3 HOPE-3 clinical trial, the experimental cell therapy deramiocel outperformed a placebo at improving arm function…
Articles by Marisa Wexler, MS
Upsher-Smith Laboratories is launching a new corticosteroid treatment for people with Duchenne muscular dystrophy (DMD) in the U.S.
A regimen of three immune-suppressing medications may improve the effectiveness of gene therapy for people with Duchenne muscular dystrophy…
A Phase 1/2 clinical trial testing SRP-1003, a treatment for myotonic dystrophy type 1 (DM1), is progressing as planned…
New findings from research in mice are challenging longstanding beliefs about the causes of Duchenne muscular dystrophy (DMD), with evidence showing…
The U.S. Food and Drug Administration (FDA) has approved an update to the prescribing information for Duchenne muscular dystrophy…
Agamree (vamorolone), a type of corticosteroid approved to help preserve muscle function in people with Duchenne muscular dystrophy…
Exon-skipping therapies Amondys 45 (casimersen) and Vyondys 53 (golodirsen) show “positive and encouraging trends” for people with Duchenne…
Note: This story was updated Nov. 5, 2025, to clarify that enrollment in the follow-up study is ongoing. Dosing has…
In a deal worth roughly $12 billion, pharmaceutical giant Novartis has agreed to acquire Avidity Biosciences, to develop…