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Washington DC - Almost six years ago, Fatiha and Abellatif Abderraziq stood in the parking lot of Washington children’s ...
Based on the late-stage data, BioMarin plans to submit a regulatory filing for expanding the label of its PKU drug Palynziq in adolescents before this year's end.
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) released topline data from the Phase 3 PEGASUS trial evaluating Palynziq ...
Newborn screening offers every child a safer, healthier start by enabling early detection, timely care, and improved outcomes ...
BioMarin Pharmaceutical Inc. today announced that the Phase 3 PEGASUS trial evaluating PALYNZIQ® (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically significant lowering ...
PKU is a rare, inherited metabolic disorder ... intellectual disabilities, behavioral symptoms or seizures. The primary endpoints are the changes in blood Phe concentration and characterization ...
As we focus on prioritizing preventive healthcare, genetic testing will remain the game-changer in reducing the burden of ...
Global Newborn Screening Market is projected to grow at a CAGR of ~6–8% from 2022 to 2027. This is likely to be driven by an ...