Phenylketonuria

May 18, 2018

Homology Medicines Presents Data from PKU Gene Therapy Program Demonstrating Long-Term Durability of Treatment in IND-Enabling Studies

Apr 30, 2018

Homology Medicines Announces Upcoming Presentations on its Genetic Medicines Platform at the American Society of Gene & Cell Therapy Meeting

Mar 28, 2018

European Medicines Agency (EMA) Accepts BioMarin's Marketing Application for Pegvaliase MAA for Treatment of Phenylketonuria (PKU)

Feb 28, 2018

Rare Disease Day 2018: BioMarin Launches RARE Scholars Scholarship Program

Dec 22, 2017

BioMarin Receives Anticipated Notification of PDUFA Extension for Pegvaliase Biologics License Application (BLA) to May 28, 2018

Oct 18, 2017

BioMarin Highlights Breadth of Innovative Development Pipeline at R&D Day on October 18th in New York

Sep 14, 2017

FDA Not Currently Planning to Hold Advisory Committee Meeting for BioMarin's Pegvaliase Biologics License Application (BLA)

Sep 05, 2017

BioMarin Announces 3 Platform and 15 Poster Presentations at 13th International Congress of Inborn Errors of Metabolism 2017

Aug 29, 2017

FDA Accepts BioMarin's Pegvaliase Biologics License Application (BLA) and Grants Priority Review Designation