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 ...

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) released topline data from the Phase 3 PEGASUS trial evaluating Palynziq ...

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.

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 ...

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 ...

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 ...

US biotech BioMarin Pharmaceutical (Nasdaq: BMRN) yesterday revealed that the Phase III PEGASUS trial evaluating Palynziq (pegvaliase-pqpz) met its primary efficacy endpoint, demonstrating a ...

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 ...