This page keeps track of new/upcoming treatment options for Phenylketonuria (PKU). What’s in development? What’s promising? What’s available soon? Find out below… Current topics include CNSA-001, APR-OD031, CDX-6114, HMI-102, gene therapy, and more.
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News
- FDA grants Orphan Drug Designation to APR-OD031 for treatment of phenylketonuria Apr 7, 2020
- ‘Innovative’ PKU treatment launched in UK Feb 17, 2020
- Censa Pharmaceuticals Announces CNSA-001 Met Primary and Secondary Endpoints in Phase 2 Trial in Patients with PKU – Positive results from Phase 2 trial demonstrate a statistically-significant and clinically-meaningful reduction in Phenylalanine Dec 3, 2019
- Update on the status of the PKU Patient Registry [video] Aug 1, 2019
Research
Clinical Trials
- Gene Therapy Clinical Study to Evaluate the Safety and Efficacy of HMI-102 in Adult PKU Subjects with PAH Deficiency (pheNIX) Mar 2, 2020
- Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of CDX-6114 in Phenylketonuria (PKU) Patients Feb 5, 2020
- Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU) Jan 28, 2020