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Clinical Trials Arena on MSN9d
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 ...
The Scientist6y
New Treatments for Phenylketonuria Aim to Loosen Reins on Strict Diet
Before newborn screening was introduced in the 1960s, phenylketonuria, a rare, inherited condition in which the body is unable to properly metabolize the amino acid phenylalanine, was a devastating ...
Nasdaq23d
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 ... to liberalize their diet while on sepiapterin treatment ...
Medindia9d
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 ...