Study 307-201 Active, Not Recruiting
A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600 µmol/L
Indication
Phenylketonuria (PKU)
Goal
This open label, two part study aims to establish dose and efficacy, safety, and tolerability of BMN 307
Investigational therapeutic
BMN 307
Status
Active, Not Recruiting
Study Type
Phase 1/ 2