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Otoferlin Gene

1 GoKawiil brief on this topic

Gene therapy Otarmeni restores hearing in children with OTOF-linked deafness

In the CHORD clinical trial, 24 participants aged 10 months to 16 years with a rare genetic hearing loss (DFNB9, caused by OTOF gene mutations) received a single infusion of the gene therapy Otarmeni into one or both ears. About 80% of participants showed improved hearing within 24 weeks, and case reports describe children regaining the ability to respond to sound and music after treatment.