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 Pharmaceutical has reported that the multi-centre Phase III PEGASUS trial of Palynziq (pegvaliase-pqpz) has met its ...
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.
BioMarin's Phase 3 trial shows Palynziq significantly reduces blood Phe in adolescents with PKU. The company plans a ...
Considering taking supplements to treat phenylketonuria pku? Below is a list of common natural remedies used to treat or reduce the symptoms of phenylketonuria pku. Follow the links to read common ...
BioMarin Pharmaceutical Inc. has advancements in phenylketonuria treatment. Click to learn how Palynziq's success could ...
(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN) Wednesday said its Phase 3 PEGASUS study evaluating Palynziq in adolescents aged 12-17 with phenylketonuria (PKU) met its primary goal. The study is ...
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 ...
evaluating the potential meaningful benefits of sepiapterin treatment in phenylketonuria or PKU patients. Sepiapterin has a dual mechanism of action to increase activity of the phenylalanine ...
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 ...