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Long-term neurocognitive outcomes of sapropterin in young children with phenylketonuria
2009-015844-41Phase 4Completed
- Sponsor
- BioMarin Pharmaceutical Inc.
- Start
- Jun 2013
- Modality
- Allosteric Modulators → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Phase 4, open-label, single-arm trial of sapropterin in phenylketonuria (PKU), sponsored by BioMarin Pharmaceutical Inc. Primary endpoint: Mean IQ score after 7 years of treatment. Started Jun 2013; status: completed.
02
Drugs
sapropterin
03
Conditions
phenylketonuria (PKU)
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