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

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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, 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 ...

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

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

Given their life-saving potential, newborn screenings should not be seen as optional but as a fundamental part of postnatal ...

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 ...

As we focus on prioritizing preventive healthcare, genetic testing will remain the game-changer in reducing the burden of ...

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.

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