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DT-216P2 Shows Promise in Friedreich Ataxia Trial

Design Therapeutics announces positive Phase 1/2 trial data for DT-216P2 in Friedreich ataxia, showing clinical improvements and good tolerability.

Sarah Johnson
BySarah Johnson- Senior Editor
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Design Therapeutics reported new biomarker and clinical data from its Phase 1/2 RESTORE-FA trial evaluating DT-216P2 in patients with Friedreich ataxia.

Sixteen patients completed four weeks of weekly intravenous DT-216P2 treatment. In the 1 mg/kg dose cohort, patients showed mean improvements of 6.4 points on the modified Friedreich's Ataxia Rating Scale.

DT-216P2 was well-tolerated with no serious adverse events. The company plans to provide an update on its development plans in the fourth quarter of 2026.

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