BioMarin Pharmaceutical Inc. has advancements in phenylketonuria treatment. Click to learn how Palynziq's success could expand BMRN's market in 2025.

BioMarin Pharmaceutical has reported that the multi-centre Phase III PEGASUS trial of Palynziq (pegvaliase-pqpz) has met its primary efficacy endpoint, showcasing a reduction in blood phenylalanine ...

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Untreated phenylketonuria can lead to brain damage, intellectual disabilities, behavioral symptoms or seizures. The primary endpoints are the changes in blood Phe concentration and characterization of ...

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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.

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.

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Washington DC - Almost six years ago, Fatiha and Abellatif Abderraziq stood in the parking lot of Washington children’s ...