Substrate replacement therapy

Origin Biosciences is investigating a substrate replacement therapy called fosdenopterin hydrobromide in clinical trials.

Molybdenum cofactor deficiency (MoCD) Type A is a genetic disorder that results from a mutation in the MOCS1 gene, which disrupts cyclic pyranopterin monophosphate (cPMP) synthesis, the first step in the molybdenum cofactor (MoCo) synthesis pathway. This creates a shortage of cPMP, a substance the human body needs to make MoCo.1-3

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References: 
  1. Schwahn BC et al. Lancet. 2015;386(10007):1955-1963.
  2. Veldman A et al. Pediatrics. 2010;125(5):e1249-e1254.
  3. Mechler K et al. Genet Med. 2015;17(12):965-970.

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