Jun 01, 2020
PTC Therapeutics Successfully Completes Acquisition of Censa Pharmaceuticals
May 08, 2020
Synlogic Reports First Quarter 2020 Financial Results and Provides Business Update
May 06, 2020
PTC Therapeutics to Acquire Censa Pharmaceuticals
Mar 26, 2020
FDA Grants Orphan Drug Designation to APR-OD031 for the Treatment of Phenylketonuria (PKU)
Mar 18, 2020
Pediatrician and Geneticist Dr. Harvey Levy Receives 2020 David L. Rimoin Lifetime Achievement Award in Medical Genetics from the ACMG Foundation for Genetic and Genomic Medicine
Mar 12, 2020
Synlogic Reports Fourth Quarter and Full Year 2019 Financial Results and Provides Business Update
Dec 03, 2019
Censa Pharmaceuticals Announces CNSA-001 Met Primary and Secondary Endpoints in Phase 2 Trial in Patients with PKU
Sep 26, 2019
BioMarin, Pioneer in Phenylketonuria (PKU) Therapies, Submits Clinical Trial Application (CTA) in U.K. for Investigational Gene Therapy for PKU
Jun 26, 2019
Global phenylketonuria treatment market is expected to grow with a healthy CAGR over the forecast period from 2019-2025
May 06, 2019
European Commission Approves Palynziq® (pegvaliase injection) for Treatment of Phenylketonuria (PKU) in Patients Aged 16 Years or Older
Mar 01, 2019
BioMarin Receives Positive CHMP Opinion in Europe for Palynziq® (pegvaliase Injection) for Treatment of Patients with Phenylketonuria (PKU) Aged 16 and Older
Nov 07, 2018
BioMarin Highlights Breadth of Innovative Development Pipeline at R&D Day on November 7th in New York
Nov 01, 2018
Cambrooke Therapeutics Metabolic Liquids and KetoVie Formulas Are Now OU-D Kosher Approved: The Only Manufacturer with Metabolic and Ketogenic Formula Products Certified
Nov 01, 2018
BioMarin to Highlight Breadth of Innovative Development Pipeline at R&D Day on November 7th in New York
Sep 05, 2018
BioMarin Announces 20 Poster Presentations at Society for the Study of Inborn Errors of Metabolism (SSIEM) Symposium 2018
Aug 13, 2018
Homology Medicines Reports Second Quarter 2018 Financial Results
Jul 05, 2018
Homology Medicines to Present at the National PKU Alliance Conference
Jun 28, 2018
BioMarin Announces First Recipients of RARE Scholars Scholarship Program
May 24, 2018
FDA approves a new treatment for PKU, a rare and serious genetic disease
May 24, 2018
BioMarin Receives Standard Approval for Palynziq(TM) (pegvaliase-pqpz) Injection for Treatment of Adults with Phenylketonuria (PKU), a Rare Genetic Disease