Acer Therapeutics Inc. Inc. (Nasdaq: ACER) (Acer) and its collaboration partner, Relief Therapeutics Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (Relief), announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to evaluate the efficacy and safety of ACER-001 (sodium phenylbutyrate) for the potential treatment of patients with Maple Syrup Urine Disease (MSUD).
MSUD is a rare, life-threatening metabolic disorder caused by a deficiency in an enzyme complex that metabolizes branched chain ketoacids, the breakdown products of the three branched-chain amino acids (BCAAs), leucine, valine, and isoleucine. Left untreated, MSUD leads to elevated plasma concentrations of these amino acids, which can lead to chronic and acute neurological damage, ranging from developmental delays in children, seizures, cognitive challenges and in some cases death. Currently, the only treatment option for patients with MSUD is a life-long, protein-restricted diet.
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