Phenylketonuria (PKU) is a disorder where an amino acid called phenylalanine accumulates inappropriately in the body. Phenylalanine is a protein building block the body obtains through the ...

BioMarin Pharmaceutical has reported that the multi-centre Phase III PEGASUS trial of Palynziq (pegvaliase-pqpz) has met its ...

Washington DC - Almost six years ago, Fatiha and Abellatif Abderraziq stood in the parking lot of Washington children’s ...

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.

Alltrna’s CEO Michelle Warner said that the approach allows for basket trials that can group patients with different diseases together.

Detailed study results will be presented at an upcoming medical meeting and submitted to health authorities later this year to expand Palynziq’s label to include adolescents.

Get a high-probability setup with clear entry and exit points right here. PKU is a rare, inherited metabolic disorder that ...

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 ...

Sepiapterin has a dual mechanism of action to increase activity of the phenylalanine hydroxylase enzyme in patients with PKU, a rare, inherited metabolic disease. The new data revealed that over ...

Our chief medical officer, Professor Jonathan Benger, explains the crucial role the HST programme plays in encouraging ...

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