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Otoferlin Deafness: FDA Approves Gene Therapy
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Silver Spring/Shanghai – The US Food and Drug Administration (FDA) has approved a gene therapy for otoferlin deafness, which accounts for 2-8% of all severe congenital hearing loss. The approval was based on the results of an open-label study in which the hearing of affected children improved within a few weeks (New England Journal of...).
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