BioMarin Pharmaceutical has reported that the multi-centre Phase III PEGASUS trial of Palynziq (pegvaliase-pqpz) has met its ...

Living with a rare disease PKU prevents Maimon from eating a certain amount of protein in a day. As a result, Maimon must ...

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.

Phenylketonuria (PKU ... the body cannot process phenylalanine from the diet effectively. Nerve cells within the brain are exceptionally sensitive to levels of phenylalanine, which explains ...

BioMarin's Phase 3 trial shows Palynziq significantly reduces blood Phe in adolescents with PKU. The company plans a ...

PKU is a rare genetic disease that manifests ... At the moment the main treatment strategy is to restrict phe in the diet, a lifetime commitment that often requires nutritional supplements to ...

The new data revealed that over 97 percent of trial participants demonstrated the ability to liberalize their diet while on sepiapterin ... for an individual without PKU, while maintaining control ...

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

San Rafael, California Thursday, April 3, 2025, 18:00 Hrs [IST] ...

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 PEGASUS trial evaluating PALYNZIQ® (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a statistically ...

Living with a rare disease PKU prevents Maimon from eating a certain amount of protein in a day. As a result, Maimon must follow a carefully controlled phenylalanine-restricted diet. As of today ...

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 ...