Patients in the Phearless study of BMN 307 – an adeno-associated virus (AAV) gene therapy designed to replace the phenylalanine hydroxylase (PAH) deficient in people with PKU – are getting a ...

NGGT has initiated Phase I/II clinical trials in the U.S. and China to evaluate the safety and efficacy of NGGT002 for the <a ta ...

Sepiapterin (formerly PTC923), an oral formulation of synthetic sepiapterin, has a dual mechanism of action to increase activity of the phenylalanine hydroxylase (PAH) enzyme. First, sepiapterin ...