Neurology Advisor

Palynziq Approved for Adolescents With Phenylketonuria

The FDA expands Palynziq approval to include pediatric patients aged 12 years and older with phenylketonuria and uncontrolled blood Phe levels.
Antelope Valley Press

U.S. Food and Drug Administration Approves BioMarin's PALYNZIQ® (pegvaliase-pqpz) for Adolescents 12 Years of Age and Older with Phenylketonuria (PKU)

Treatment with PALYNZIQ led to statistically significant blood phenylalanine (Phe) lowering compared to diet alone in pivotal Phase 3 PEGASUS study ...

Beam Therapeutics Reports Fourth Quarter and Year-End 2025 Financial Results and Announces New Liver-Targeted Genetic Disease Program in Phenylketonuria (PKU)

New Program Designed as Platform-based Approach for Direct Correction of Mutations Causing PKU; Investigational New Drug (IND) Filing for ...

Woman Adopts Child With Same Rare Condition: Managing PKU Together

Growing up with a rare genetic disorder, Pam Kowalczyk often felt like no one truly understood her daily struggles. Years ...
Seeking Alpha

Maze Therapeutics Announces Positive First-in-Human Results from Phase 1 Trial of MZE782, Establishing Proof of Mechanism for a Potent, Oral SLC6A19 Inhibitor with Potential to ...

Phase 1 data in healthy volunteers exceed expectations and support best-in-class potential, enabling Phase 2 advancement for both intended indications of PKU and CKD Dose-dependent urinary amino acid ...
Seeking Alpha

PTC Therapeutics Receives Positive CHMP Opinion for Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)

WARREN, N.J., April 25, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has ...