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Long-Term Follow-Up Study of Gene Therapy for AADC Deficiency (AADC-LTFU)
jRCT1033260442Recruiting
- Sponsor
- Gene Therapy Research Institute
- Start
- Aug 2026
- Countries
- 1
01
Overview
Open-label, single-arm trial in Aromatic L-amino acid decarboxylase deficiency (AADC), sponsored by Gene Therapy Research Institute. Primary endpoints: Motor function, Mental development and 3 more. Started Aug 2026; status: recruiting.
02
Conditions
Aromatic L-amino acid decarboxylase deficiency (AADC)
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