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EPIPHENY long-term neurocognitive outcomes study in pediatric phenylketonuria
2024-514435-20-00Phase 3Authorised
- Sponsor
- PTC Therapeutics, Inc.
- Start
- Dec 2025
- Modality
- Monoclonal Antibodies → Antibody-Based Therapeutics
- Countries
- 8
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Overview
Phase 3, open-label, single-arm trial of adalimumab in phenylketonuria (PKU), sponsored by PTC Therapeutics, Inc. Primary endpoint: Mean change in full-scale intelligence quotient over 2 years. Planned start Dec 2025; status: authorised.
02
Drugs
03
Conditions
phenylketonuria (PKU)
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