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Gene Therapy Trial for Aromatic L-Amino Acid Decarboxylase Deficiency
2019-003072-39Phase 2
- Sponsor
- National Taiwan University Hospital
- Start
- Oct 2019
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of ezaladcigene resoparvovec in Aromatic L-amino acid decarboxylase deficiency (AADC), sponsored by National Taiwan University Hospital. Primary endpoint: Increase From Baseline in Neurotransmitter Metabolites (HVA or 5-HIAA).
02
Drugs
ezaladcigene resoparvovec
03
Conditions
Aromatic L-amino acid decarboxylase deficiency (AADC)
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