/https://tf-cmsv2-smithsonianmag-media.s3.amazonaws.com/filer_public/cf/cd/cfcd970f-b205-4a58-b5c2-3c656d99b432/baby-hearing-test.jpg)
The FDA Approves the First-Ever Gene Therapy for Deafness, Which Aims to Restore Hearing in Kids With a Rare Inherited Condition
Key takeaways
- Undiagnosed issues can delay developmental milestones.
- The therapy, called Otarmeni, is approved for the treatment of otoferlin deafness, a type of genetic hearing loss that affects an estimated 50 babies born each year in the United States.
- This form of deafness is caused by a mutated gene that encodes a faulty protein called otoferlin.
Undiagnosed issues can delay developmental milestones. BSIP / UIG Via Getty Images On April 23, the Food and Drug Administration approved the first-ever gene therapy for a rare, congenital form of deafness. The treatment’s maker, biotechnology company Regeneron, says it plans to provide the therapy for free to patients in the United States.
“This really is life-changing for families with children with hearing loss,” says Eliot Shearer, a pediatric otolaryngologist at Boston Children’s Hospital, who helped lead a clinical trial investigating the treatment, to Berkeley Lovelace Jr. at NBC News.
The therapy, called Otarmeni, is approved for the treatment of otoferlin deafness, a type of genetic hearing loss that affects an estimated 50 babies born each year in the United States. It accounts for about 2 percent to 8 percent of people with inherited deafness.