A Phase 1/2 clinical trial testing SRP-1003, a treatment for myotonic dystrophy type 1 (DM1), is progressing as planned…
Articles by Marisa Wexler, MS
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…
Use of the oral therapy BBP-418 appeared to improve walking ability and lung function in people with limb-girdle muscular…
Three boys with Duchenne muscular dystrophy (DMD) who were given the experimental gene therapy GNT0004 in a clinical trial…
Health Canada has approved Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients ages 4 and older,…