Jun 01, 2020
PTC Therapeutics Successfully Completes Acquisition of Censa Pharmaceuticals
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)
Feb 21, 2020
FDA Authorizes Marketing of the First Genetic Test to Aid in the Diagnosis of Fragile X Syndrome
Jan 27, 2020
Fragile X Syndrome (FXS): Current Treatment Practices, Emerging Drugs, Market Share of Individual Therapies, Current & Forecasted Market Size from 2017 to 2028 Segmented by 7 Major Markets
Jan 14, 2020
Therap's Tools Help Agencies Supporting Individuals with Intellectual and Developmental Disabilities Improve their Documentation and Communication
Dec 03, 2019
Censa Pharmaceuticals Announces CNSA-001 Met Primary and Secondary Endpoints in Phase 2 Trial in Patients with PKU
Nov 14, 2019
Poised for Significant Growth and Profitability, BioMarin Shares Company Highlights During R&D Day on November 14th in New York
Jun 27, 2019
Fragile X Syndrome Market Spotlight, 2019 - There Have Been 5 Licensing & Asset Acquisition Deals Involving FXS Drugs During 2014-2019
May 06, 2019
European Commission Approves Palynziq® (pegvaliase injection) for Treatment of Phenylketonuria (PKU) in Patients Aged 16 Years or Older
Apr 30, 2019
TSC Global Awareness Day Scheduled for May 15
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
Dec 05, 2018
Therap Announces 2019 National Conference Keynote Speakers
Nov 29, 2018
Tuberous Sclerosis Alliance Preclinical Consortium Announces New Staff and Company Members
Nov 07, 2018
BioMarin Highlights Breadth of Innovative Development Pipeline at R&D Day on November 7th in New York
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
May 18, 2018
Homology Medicines Presents Data from PKU Gene Therapy Program Demonstrating Long-Term Durability of Treatment in IND-Enabling Studies