Tag Archive for: sepiapterin

The goal of the trial was to reduce levels of phenylalanine, an amino acid, in the blood. Patients with the inherited metabolic disorder cannot break down phenylalanine on their own, causing a build-up of the acid, which often leads to memory and neurological issues.  

PTC Therapeutics reported Q4 and full-year 2022 results Tuesday ahead of a pivotal 2023 featuring three late-stage readouts in registration-directed trials.