One in 25,000 people in the U.S. are diagnosed with PKU, a genetic disorder that prevents to body from breaking down an amino ...

The US Food and Drug Administration (FDA) has approved biotech BioMarin’s Palynziq (pegvaliase-pqpz) for adults with the rare and serious genetic disease phenylketonuria (PKU). Patients with PKU ...

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down phenylalanine (phe), an amino acid that is commonly found in many foods. Left untreated ...

"Genetic counseling is the process of helping ... genetics professionals have the potential to prevent newborns with phenylketonuria – a genetic condition known as PKU – from becoming gravely ...

The drug is currently the only enzyme therapy to treat the disease in this age group. PKU is a rare genetic disorder that prevents the body from breaking down Phe, an amino acid found in protein ...

Rare genetic variations in the phenyalanine hydroxylase gene are primarily responsible for the condition. [9] Individuals with PKU can develop neurological damage from excess phenylalanine [10 ...

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 ...

MZE829 Phase 2 HORIZON Trial Enrolling Patients with APOL1 Kidney Disease (AKD); Initial Data Expected in Q1 2026MZE782 Phase 1 Healthy Volunteer ...

India has a notable prevalence of genetic disorders, such as sickle cell anemia, thalassemia, cystic fibrosis, and phenylketonuria (PKU). Given the widespread occurrence of these conditions ...

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 ...