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 primary efficacy endpoint, showcasing a reduction in blood phenylalanine ...

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.

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 ...

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 expand BMRN's market in 2025.

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] ...

SAN RAFAEL, CA, USA I April 2, 2025 I BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the Phase 3 PEGASUS trial evaluating PALYNZIQ ® (pegvaliase-pqpz) met its primary efficacy ...