ACMG’s latest Therapeutics Bulletin highlights Kebilidi (eladocagene exuparvovec-tneq), the first FDA-approved gene therapy for AADC deficiency and the first FDA-approved intraputaminal gene therapy of any kind. Read more in GIM Open: tinyurl.com/3rva2kkd #Therapeutics #AADCd #RareDisease
August 6, 2026 at 3:47 PM
ACMG’s latest Therapeutics Bulletin highlights Kebilidi (eladocagene exuparvovec-tneq), the first FDA-approved gene therapy for AADC deficiency and the first FDA-approved intraputaminal gene therapy of any kind. Read more in GIM Open: tinyurl.com/3rva2kkd #Therapeutics #AADCd #RareDisease