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Design Therapeutics Announces Four-Week IV Data from the RESTORE-FA Trial of DT-216P2 Demonstrating Clinical Improvements and Comprehensive Biomarker Activity in Friedreich Ataxia
GlobeNewswire Inc.·
Design Therapeutics announced positive Phase 1/2 data from its RESTORE-FA trial showing DT-216P2 demonstrated dose-dependent clinical improvements in Friedreich ataxia patients after four weeks of treatment, including improvements in mFARS scores, upright stability, and fatigue. The drug showed comprehensive biomarker activity with 65% increase in FXN mRNA and 22-27% increase in FXN protein levels. The company plans to advance toward registrational development with updates expected in Q4 2026.
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