Gene Therapy Restores Hearing in UK Toddler Born Deaf

Published on Spencer Knight via LinkedIn

Regeneron has announced a major milestone in pediatric gene therapy using its DB-OTO treatment. In a trial involving 11 children with congenital deafness, aged 10 months to 16 years, the therapy restored hearing levels across most speech-relevant frequencies.

One of the youngest successes is 18-month-old Opal Sandy, born completely deaf. After receiving the one-time treatment, she now hears whispers and has begun speaking her first words, like “Dada.” Her progress highlights the potential of targeted gene therapies to change lives, especially in rare diseases.

Read the full article via BBC News:

👉 Pioneering gene therapy restores UK girl’s hearing

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