Sarepta Therapeutics Advances SRP-1003 Myotonic Dystrophy Trial to Higher Dose Cohorts

Reuters11-24
<a href="https://laohu8.com/S/SRPT">Sarepta Therapeutics</a> Advances SRP-1003 Myotonic Dystrophy Trial to Higher Dose Cohorts

Sarepta Therapeutics Inc. has provided an update on its Phase 1/2 multiple ascending dose clinical study of SRP-1003, an investigational small interfering RNA (siRNA) therapeutic for the treatment of type 1 myotonic dystrophy (DM1). Cohorts 1 (1.5 mg/kg) and 2 (3 mg/kg) have been completed, while cohort 3 (4.5 mg/kg) is fully enrolled and ongoing. Following a positive pre-specified drug safety committee review, the study is advancing with additional dose escalation cohorts. Patients are currently being dosed in cohort 4 (6 mg/kg), with plans to initiate dosing in the final cohort (12 mg/kg) in early 2026. Study results have not yet been presented.

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