FDA ApprovedUnited States
First FDA-Approved Gene Therapy for AADC Deficiency
Kebilidi (eladocagene exuparvovec-tneq) — one-time intraputaminal gene therapy for aromatic L-amino acid decarboxylase deficiency
FDA accelerated approval on 13 November 2024 (press 14 November 2024) for Kebilidi (eladocagene exuparvovec-tneq) suspension — an AAV…
This is for families of children (and adults) with genetically confirmed AADC deficiency from biallelic…