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ACER-001

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Company Acer Therapeutics Inc.
Description Taste-masked, immediate-release formulation of sodium phenylbutyrate (NaPBA)
Molecular Target Branched chain ketoacid dehydrogenase kinase (BCKDK) (BDK)
Mechanism of Action 
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentPhase II
Standard IndicationUrea cycle disorder (UCD)
Indication DetailsTreat maple syrup urine disease (MSUD); Treat urea cycle disorder (UCD)
Regulatory Designation U.S. - Orphan Drug (Treat maple syrup urine disease (MSUD))
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