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 ...
pharmaphorum2y
FDA slaps clinical hold on BioMarin's PKU gene therapy
One mutation eliminated the PAH gene that's missing in PKU, while the second rendered the animals immunodeficient, which could also raise the risk of malignancy.
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] ...
Hosted on MSN29d
Key Indicators Of Phenylketonuria
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 Seeks Label Expansion For Palynziq After Positive Phase 3 Results In Adolescent Patients
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) released topline data from the Phase 3 PEGASUS trial evaluating Palynziq ...
BioMarin Pharmaceutical: Pegasus Success May Lead To Palynziq Label Expansion
BioMarin Pharmaceutical Inc. has advancements in phenylketonuria treatment. Click to learn how Palynziq's success could ...
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 ...
Nature6y
Chaperones to the rescue
Protein misfolding is a common feature of genetic diseases such as cystic fibrosis and phenylketonuria (PKU). For these diseases, compounds that aid the folding and/or increase the native-state ...