Current Studies
Study 307-201
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
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Indication
Phenylketonuria (PKU)
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Investigational Therapeutic
BMN 307
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Study Type
Phase 1/ 2
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Goal
This open label, two part study aims to establish dose and efficacy, safety, and tolerability of BMN 307
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Status
Active, Not Recruiting
Study 307-902
A Prospective Clinical Study of Phenylketonuria (PKU)
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Indication
Phenylketonuria (PKU)
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Study Type
Observational
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Goal
The objective of this study is to collect longitudinal data assessing markers of disease and clinical outcomes in PKU subjects ≥ 14 years old.
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Status
Active, Not Recruiting