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Regeneron wins FDA approval for first gene therapy for genetic hearing loss
Clinical trial data showed 90% of participants improved, and some children later learned to speak, researchers said.
- Researchers reported Wednesday in the journal Nature that experimental gene therapy successfully restored hearing in people born with a rare form of deafness caused by OTOF gene mutations.
- The OTOF gene encodes otoferlin, a protein critical for translating sound vibrations into electrical signals the brain can interpret; without it, children are born completely deaf.
- About 90 percent of recipients saw hearing improvements, with the study involving participants aged nine months to 32 years and reporting no serious side effects or dose-related toxicity.
- Recipients showed improved speech perception, with some children learning to speak for the first time; gene therapy may eventually prove superior to mechanical Cochlear implants requiring maintenance.
- Regeneron Pharmaceuticals plans to file for regulatory approval, potentially becoming the first gene therapy for deafness to clear the Food and Drug Administration within the next year.
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FDA approves first gene therapy for inherited deafness, shown to restore hearing for children with rare condition
The US Food and Drug Administration on Thursday approved the first gene therapy for inherited hearing loss, a one-time treatment that proved to be life-changing for a small number of children in a clinical trial who were born without hearing.
·Atlanta, United States
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Total News Sources24
Leaning Left4Leaning Right1Center19Last UpdatedBias Distribution79% Center
Bias Distribution
- 79% of the sources are Center
79% Center
L 17%
C 79%
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