Programme SOM1311 for the Treatment of Phenylketonuria
SOM1311 is a small molecule pharmacological chaperone of Phenylalanine Hydroxylase for the treatment of Phenylketonuria.
Phenylketonuria (PKU) is characterised by an inborn error of metabolism that results in decreased metabolism of the amino acid phenylalanine. Untreated, PKU can lead to intellectual disability, seizures, behavioural problems and mental disorders.
