Gene Therapy Improves Auditory Response for Child With Profound Genetic Hearing Loss
A child with profound genetic hearing loss was dosed with the investigational otoferlin gene therapy (DB-OTO), becoming the first patient dosed in a phase 1/2 trial evaluating the treatment, and experienced improved auditory response at week 6 with no concerning safety signals.
United Kingdom United States Regeneron Pharmaceuticals Cambridge University Hospitals Contemporary Pediatrics Image Credit
Source: ajmc.com