The company is advancing a robust pipeline of ENB-based therapeutics targeting both rare and prevalent chronic diseases, ...

The U.S. Food and Drug Administration has approved Sephience (sepiapterin), an oral therapy by PTC Therapeutics, for the treatment of phenylketonuria (PKU), a rare inherited metabolic disorder that ...

When Conor Byrne got the call from the hospital just days after his son Jacob was born, he knew something wasn’t right. “Don’t Google it,” they were told — words that no new parent ever wants to hear.

Phase 1 data in healthy volunteers exceed expectations and support best-in-class potential, enabling Phase 2 advancement for both intended indications of PKU and CKD Dose-dependent urinary amino acid ...