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Although these ADAR-based, RNA editing therapeutics are highly specific to making A to I modifications in mRNA transcripts, they still provide a promising alternative therapeutic route to clinical translation of NDD therapeutics

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Cycle guidelines Cycle guidelines Approach Phase 2 reference Duration 48 weeks Review Point Every 4 weeks at each dose step Best For Matches the original NEJM trial structure Approach Phase 3 TRIUMPH reference Duration 68-72 weeks Review Point Every 4 weeks