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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