Regeneron Pharmaceuticals · Gene Therapy · Approved
ApprovedTherapeuticStronger sources
Population
Children & adults · FDA label has no age bounds; trial: 10 months–16 years
Biallelic OTOF severe-to-profound deafness
Evidence
regulatory
Confidence
99%
Indications
Hearing LossSensorineural Hearing LossGenetic Hearing LossOTOF-Related Hearing Loss



