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Long-term observational study of Gene Therapy for AADC deficiency
jRCT2033220245Active, not recruiting
- Sponsor
- Gene Therapy Research Institute
- Start
- Aug 2022
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Open-label, single-arm trial of ezaladcigene resoparvovec in Aromatic L-amino acid decarboxylase deficiency (AADC), sponsored by Gene Therapy Research Institute. Primary endpoints: Long-term tolerability, Long-term safety and 1 more. Started Aug 2022; status: active, not recruiting.
02
Drugs
ezaladcigene resoparvovec
03
Conditions
Aromatic L-amino acid decarboxylase deficiency (AADC)
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