Две сестры из Висконсина стали первыми в США, получившими одобренную терапию генами от редкой глухоты

Sisters await results after gene therapy for rare hearing loss GREEN BAY, Wis. (WBAY) - Two sisters from northeast Wisconsin were the first children in the country to receive a newly FDA-approved gene therapy for a rare form of hearing loss outside of a clinical trial. Diagnosis takes time Cleo and Lennon are sisters, about three and a year-and-a-half old. Both have the same rare genetic form of hearing loss. For their parents, Ty and Emily, figuring that out took time. “After some further evaluation, we were told that she could hear. And then kind of just as time progressed on, we just noticed, like, vocabulary. It just wasn’t coming quickly. Only a few words here and there,” Emily said. That was Cleo. Then Lennon also failed her newborn hearing screening. Eventually, genetic testing gave them an answer. “But, so then we found out, once we did find out that it was the OTOF gene, the Otoferlin gene, then we were, like, pretty much trying as hard as we could to get them in a trial,” Ty said. How the therapy works The girls were missing the genetic code needed to help the inner ear make a protein needed to pass sound signals to the brain. Children’s Wisconsin has already been studying a gene therapy designed to replace what’s missing. “And this genetic therapy delivers that missing bit of genetic code to the inner ear so that it can manufacture that protein and reestablish that continuity. And so the children can hear,” said Dr. Michael Harris. The therapy is called otarmeni. For families like this one, it changes a conversation doctors have been having for years. “Now with this treatment option, we’re able to actually offer a treatment for the hearing loss and not just manage it,” said Dr. Brittney Gniedziejko, an audiologist. Procedure and aftermath When the FDA approved the therapy, both girls underwent the procedure at Children’s Wisconsin, making Cleo and Lennon the first children in the country to receive the FDA-approved therapy outside of a clinical trial. “There was just a lot of emotions.... emotions all around,” Emily said. “I think we were kind of excited that we were finally at a place where we were doing something. Most of it was just test, test, test,” Ty said. The procedure is over. Now comes the wait. “And patience is now one of the biggest things,” Ty said. What comes next Doctors say the biggest improvements in hearing typically come about six months after surgery. At home, their parents say they’re already noticing some new responses from Cleo. “Like, I’ll come into a room and I’ll clap, and then she’ll, like, whip her head now, where she wasn’t really necessarily doing that before,” Ty said. More testing over the next several months will give the family a better idea of how well the treatment worked. Copyright 2026 WBAY. All rights reserved.