Posted innews Otorhinolaryngology Pediatrics
AAV‑OTOF Gene Therapy Restores Substantial Hearing in DFNB9 Across Ages — Rapid, Age‑dependent Benefits in a First-in‑Human Trial
A single‑arm trial of AAV‑OTOF (Anc80L65) in 10 patients (1.5–23.9 years) with autosomal recessive deafness 9 showed good tolerability and rapid, clinically meaningful hearing gains, with optimal outcomes in children aged 5–8 years; longer follow‑up is required.