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

A rare disease in the US is one in 1,500, or fewer than 200,000 people, and an ultra-rare disease is one in 50,000 or fewer ...

BioMarin BMRN has announced positive results from the phase III PEGASUS study evaluating Palynziq (pegvaliase) in adolescents aged 12-17 years with phenylketonuria (PKU). The study achieved its ...

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.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) released topline data from the Phase 3 PEGASUS trial evaluating Palynziq ...

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

Synthetic biology specialist Synlogic will start phase 3 testing of a drug for rare metabolic disorder phenylketonuria (PKU) next year, after reporting solid proof-of-concept data.

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

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 ...

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

Maimon was diagnosed with PKU at birth. PKU is a genetic disorder that causes the amino acid phenylalanine to build up in the body. Both parents had to carry the gene for PKU in order for Maimon ...