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28 note to investors, William Blair analysts wrote that Applied Therapeutics suspects the CRL was related to the efficacy package as opposed to [chemistry, manufacturing and controls] or safety. Results from the Phase III ACTION-Galactosemia Kids study, unveiled in April 2023, showed that govorestat fell short of its primary efficacy endpoint, a composite of four clinical measures designed to assess various domains of galactosemia symptoms

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Reminder The pathways above describe compound-level research and mechanism

While nearly all this research involves animal models, the consistency of results across different injury types and body systems has generated significant interest in the peptide therapy community