A claims-based study revealed high adherence to phosphorodiamidate morpholino oligomer (PMO) treatments for Duchenne muscular dystrophy (DMD), but highlights limitations in interpreting real-world effectiveness from claims data.

Boy with DMD using walker | Image credit: Svitlana – stock.adobe.com

Viltolarsen Slows DMD Progression in Small Study

By Hayden E. Klein

November 23rd 2024

Further research is needed to determine how once-weekly viltolarsen can slow disease progression and preserve motor function in boys and young men with Duchenne muscular dystrophy (DMD).

FDA website | Image Credit: © Postmodern Studio - stock.adobe.com

FDA Accepts Resubmitted NDA for Ataluren in Nonsense Duchenne Muscular Dystrophy

By Marco Meglio

November 22nd 2024

The new drug application (NDA) includes data from a global placebo-controlled, 72-week study as well as findings from the STRIDE registry, an ongoing, observational, real-world study of ataluren in routine care.

Researcher working with microplate in the laboratory | Image credit: angellodeco – stock.adobe.com

Sarepta Halts Development, Testing of DMD Therapy SRP-5051

By Hayden E. Klein

November 14th 2024

Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular dystrophy (DMD) was stopped, with the developer citing safety concerns, FDA feedback, and an evolving therapeutic landscape.

Gene therapy research | Image credit: RFBSIP – stock.adobe.com

A Year of DMD Gene Therapy Trial Failures

By Hayden E. Klein

October 29th 2024

The outcomes of the EMBARK and CIFFREO trials in boys aged 4 to 7 have raised concerns in the Duchenne muscular dystrophy (DMD) community and prompted layoffs at Pfizer.

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