biotech / news / / IFLScience
Congenital hearing loss can be traced back to a genetic mutation in about half of cases.
The FDA has approved Otarmeni, a gene therapy for OTOF-related genetic hearing loss.
KEY POINTS
- Otarmeni uses two viral vectors to deliver the large OTOF gene into inner ear hair cells.
- Otarmeni is available free to eligible US patients without cochlear implants and severe hearing loss.
- Initial trial results showed 80 percent of treated children experienced hearing improvement within 24 weeks.
- Five of twelve patients followed up to 48 weeks achieved normal hearing, including the ability to hear whispers.
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