Dossier
Otoferlin
Coverage of Otoferlin in the Nexus archive.
- First-ever gene therapy for hearing loss gets FDA approval: 'Groundbreaking'
The FDA approved Otarmeni, the first dual adeno-associated virus (AAV) gene therapy for genetic hearing loss linked to the OTOF gene. The treatment, administered surgically to the inner ear, improved hearing in some patients with OTOF-related deafness, marking the first disease-modifying therapy for this condition.