At a February 2025 meeting,researchers presented their latest findings from a phase 1/2 clinical trial of a gene therapy,DB-OTO,developed by Regeneron Pharmaceuticals(Tarrytown,NY,USA)for a particular type of profound...At a February 2025 meeting,researchers presented their latest findings from a phase 1/2 clinical trial of a gene therapy,DB-OTO,developed by Regeneron Pharmaceuticals(Tarrytown,NY,USA)for a particular type of profound hearing loss in children.Nearly all the 12 participants,with no serious adverse events,experienced clinically meaningful and durable hearing improve-ments[1].A toddler born deaf,for example,could hear well enough after treatment to quack when asked,“What sound does a duck make?”[2].展开更多
文摘At a February 2025 meeting,researchers presented their latest findings from a phase 1/2 clinical trial of a gene therapy,DB-OTO,developed by Regeneron Pharmaceuticals(Tarrytown,NY,USA)for a particular type of profound hearing loss in children.Nearly all the 12 participants,with no serious adverse events,experienced clinically meaningful and durable hearing improve-ments[1].A toddler born deaf,for example,could hear well enough after treatment to quack when asked,“What sound does a duck make?”[2].