"This study will investigate the safety and tolerability of treatment with oral administration of NNZ-2566 at 35 mg/kg or 70 mg/kg BID in adolescent or adult males with Fragile X Syndrome. The study also will also investigate measures of efficacy during treatment."
From the clinical trials website. Presumably this wasn't powered for efficacy - therefore, is very nice to see the dose-dependent results reach statistical signifcance as likely means a larger biological effect than oringinally thought. Anyone have the trial design specifics with stats in it?????
Ann: Trofinetide successful in Phase 2 trial in Fragile X, page-24
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