Cycle Pharmaceuticals (Cycle) has launched Cycle Vita PKU, an AI-powered app to help patients with PKU manage every aspect of ...

Also known as PAH deficiency, phenylketonuria is a genetic condition. Credit: bangoland / Shutterstock. BioMarin Pharmaceutical has reported that the multi-centre Phase III PEGASUS trial of Palynziq ...

PEGASUS is a Phase 3 multi-center open-label randomized controlled study evaluating the safety and efficacy of PALYNZIQ compared to diet alone in 55 adolescents aged 12-17 with phenylketonuria.

evaluating the potential meaningful benefits of sepiapterin treatment in phenylketonuria or PKU patients. Sepiapterin has a dual mechanism of action to increase activity of the phenylalanine ...

The $800 million deal hands Otsuka a drug that’s shown promise in early testing in phenylketonuria, and is the fourth buyout of a privately held biotech in the last two weeks alone. Otsuka is focused ...

Phenylketonuria (PKU) is a recessive disorder of phenylalanine metabolism due to mutations in the gene for phenylalanine hydroxylase (PAH). Reduced PAH activity results in significant ...