“I was testing her with the implant in place and she erupted into pretty loud applause, not realizing that the implant was actually out. When she turned around for the first time, I couldn’t believe it. I couldn’t.
“I thought it was a fluke or a change in the light or something that caught her eye, but I repeated it a few times. It really freaked me out.”
Opal, who underwent surgery at Addenbrookes Hospital in Cambridge, is one of up to 18 children to take part in the trial, which is being carried out in the UK, US and Spain.
Opal received a low dose in one ear, but with more evidence that it is safe, some children may receive a higher dose in the other ear before receiving treatment in both ears. doses will be administered.
Professor Manohar Vance, an ear surgeon at Cambridge University Hospitals NHS Foundation Trust, which runs Adevenbrooks, and lead investigator on the trial, said the results after 24 weeks were “better than I expected”. Stated.
“We hope that it becomes a potential treatment.”
“We achieved the following results. [Opal] This is very impressive and comes very close to restoring normal hearing. Therefore, we are hopeful that it may be a potential treatment.
“She has pretty much normal hearing for her age in terms of being able to hear small sounds (like whispers),” he said. “I don’t know if she can understand words the same way because she’s too young, but I’m sure she can respond to small sounds.”
A second child is also undergoing gene therapy at Cambridge University Hospital, with positive results recently seen six weeks after surgery, while other gene treatments for hearing loss are being tested in China and the United States.
Vance estimates that around 20,000 people in the US, Germany, France, Italy, Spain and the UK have auditory neuropathy caused by OTOF mutations.
“This trial will help us learn more about the effectiveness of gene therapy in cases where hearing loss has a specific genetic cause,” said Martin McLean, senior policy advisor at the National Association for Deaf Children.
“I want to emphasize that with the right support from the start, hearing loss should never stand in the way of happiness and fulfillment.”
Sisters compete to see how much noise they can make
Sandi said Opal has started talking in the past six weeks, and both girls now like to “see who can make the most noise.”
“She’s good at all the common first words for babies, so her favorites are ‘daddy,’ ‘umm, aah,’ and ‘bye-bye,'” she said.
“I always said I never get annoyed when they make noise, but I do get annoyed when they make noise,” Mrs. Sandy joked.
“Opal loves playing with little sets of instruments…beating the drums, playing the little piano, banging on wooden blocks, things like that.
“Nora has only recently become interested in music, and[Opal]likes to raise her arms and do a little dance in the kitchen.
“That’s why they like to dance together. Nora likes to read to her, she likes to fight, she likes to jump off the couch.”
The results of the Chord trial were presented on Wednesday at the American Society for Gene and Cell Therapy meeting in Baltimore, USA.