Pharmacologic inhibition of an upstream enzyme to reduce accumulation of toxic metabolites has been successfully used to treat other inherited metabolic disorders (IMDs), most notably tyrosinaemia type I.11 Theoretically, such an approach would reduce the accumulation of neurotoxic metabolites in PDE-ALDH7A1 and provide an alternative to nutritional therapies. This evidence concerns the gene ALDH7A1 and inborn errors of metabolism.