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Current Studies


 

Study 307-201

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)

  • Investigational Therapeutic

    BMN 307

  • Study Type

    Phase 1/ 2

  • Goal

    This open label, two part study aims to establish dose and efficacy, safety, and tolerability of BMN 307

  • Status

    This study is currently recruiting participants

View Study 307-201 Now
 

Study 307-902

Recruiting

A Prospective Clinical Study of Phenylketonuria (PKU)

  • Indication

    Phenylketonuria (PKU)

  • Study Type

    Observational

  • Goal

    The objective of this study is to collect longitudinal data assessing markers of disease and clinical outcomes in PKU subjects ≥ 14 years old.

  • Status

    This study is currently recruiting participants

Visit Study 307-902 Now