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Dyne Therapeutics Initiates Phase 3 Trial of Genetic Neuromuscular Disease Treatment
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Dyne Therapeutics Initiates Phase 3 Trial of Genetic Neuromuscular Disease Treatment
Mar 11, 2026 6:56 AM

04:30 AM EDT, 03/09/2026 (MT Newswires) -- Dyne Therapeutics ( DYN ) said over the weekend it started a phase 3 study of zeleciment basivarsen in participants with myotonic dystrophy type 1, a rare genetic neuromuscular disease.

The 48-week study will enroll about 150 patients aged at least 16 years, who will be given 6.8 mg/kg of z-basivarsen or placebo every eight weeks, to evaluate the primary endpoint in the change from baseline in the five times sit to stand test at week 49.

Separately, the company said data from a current phase 1/2 clinical study of zeleciment rostudirsen in participants with Duchenne muscular dystrophy amenable to exon 51 skipping demonstrated improvements from baseline in lung function and left ventricular ejection fraction.

The results compared with the expected decline estimated in published natural history data for both indications, according to Dyne Therapeutics ( DYN ).

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