Design Therapeutics, Inc. - 8-K Filing
Design Therapeutics announced positive clinical data from the Phase 1/2 RESTORE-FA trial of DT-216P2 in Friedreich ataxia patients as of May 17, 2026. Patients receiving the highest dose (1 mpk) showed mean improvements of 6.4 points on the modified Friedreich’s Ataxia Rating Scale and 2.7 points on Upright Stability Score after four weeks of treatment. Biomarker results included a 65% increase in whole blood FXN mRNA levels, with no serious adverse events reported. (Items: 8.01 Other Events)
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