One mutation eliminated the PAH gene that's missing in PKU, while the second rendered the animals immunodeficient, which could also raise the risk of malignancy. Of 63 animals treated, six of ...

Pegvaliase statistically significantly lowered blood Phe levels in adolescents with PKU compared with diet alone. Topline data were announced from a phase 3 trial evaluating pegvaliase-pqpz in ...

These specific mutations are found across a range of ... methylmalonic acidaemia (MMA) and phenylketonuria (PKU). One of the ...

The PKU-associated enzyme deficiency was determined ... to potentially "cancel out" the negative effects of X-linked mutations, they are far more likely than women to be affected by X chromosome ...

PALYNZIQ is the first and only enzyme therapy approved to treat adults with PKU. BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.) Detailed results from the PEGASUS study will ...

Cycle Pharmaceuticals (Cycle) has launched Cycle Vita PKU, an AI-powered app to help patients with PKU manage every aspect of their treatment plan. It can be challenging for patients with PKU ...