Clinical Trials Arena on MSN1d
BioMarin’s trial for phenylketonuria therapy meets primary efficacy endpoint
BioMarin Pharmaceutical has reported that the multi-centre Phase III PEGASUS trial of Palynziq (pegvaliase-pqpz) has met its ...
BioMarin Seeks Label Expansion For Palynziq After Positive Phase 3 Results In Adolescent Patients
BioMarin's Phase 3 trial shows Palynziq significantly reduces blood Phe in adolescents with PKU. The company plans a ...
Pharmabiz1d
BioMarin Pharma’s phase 3 PEGASUS trial evaluating Palynziq in adolescents with phenylketonuria meets primary efficacy endpoint
San Rafael, California Thursday, April 3, 2025, 18:00 Hrs [IST] ...
Nasdaq15d
PTC Therapeutics' New Data Shows Meaningful Benefit Of Sepiapterin In Phenylketonuria Patients
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 ...
Medindia1d
BioMarin Announces Positive Pivotal Data for PALYNZIQ (pegvaliase-pqpz) in Adolescents with Phenylketonuria
PR Newswire SAN RAFAEL, Calif., April 2, 2025 Company plans to submit for regulatory approval later this year SAN RAFAEL, Calif., April 2, 2025 /PRNewswire/ -- BioMarin Pharmaceut ...
Monthly Prescribing Reference2y
Javygtor Powder
Sapropterin dihydrochloride 100mg; per pkt; pwd for oral soln. Javygtor is a synthetic form of BH4, the cofactor for the enzyme phenylalanine hydroxylase (PAH). PAH hydroxylates Phe through an ...