Boy with Hunter syndrome makes historic recovery after world-first gene therapy
A three-year-old boy, Oliver Chu, becomes the first patient in the world to receive a revolutionary gene therapy for Hunter syndrome, a rare genetic disorder. After the groundbreaking treatment in Manchester, doctors report astonishing progress in his physical and cognitive development, offering new hope for children with rare diseases.
A three-year-old boy has amazed doctors after becoming the first person in the world with his devastating condition to receive a groundbreaking gene therapy, News.Az reports, citing BBC.
Oliver Chu suffers from Hunter syndrome (MPS II), a rare inherited disorder that gradually damages the body and brain. In severe cases, patients rarely live past 20, and symptoms are sometimes described as a form of childhood dementia.
Before the treatment, Oliver could not produce a vital enzyme necessary for healthy cellular function due to a faulty gene.
Medical experts in Manchester attempted to halt the disease by altering Oliver’s cells through gene therapy—a global first.
“I’ve been waiting 20 years to see a boy like Ollie doing as well as he is, and it’s just so exciting,” said Prof Simon Jones, co-leader of the trial.
Oliver is the first of five boys worldwide to receive this treatment, and a year later, he is showing signs of normal development.
“Every time we talk about it I want to cry because it’s just so amazing,” says his mother, Jingru Chu.
Oliver and his father, Ricky, first visited the Royal Manchester Children’s Hospital in December 2024. Since his diagnosis in April, Oliver’s life—like that of his older brother Skyler, who also has the condition—had been dominated by hospital visits.
Children with Hunter syndrome often appear healthy at birth but show symptoms around age two. These can include physical changes, limb stiffness, short stature, and damage to the heart, liver, bones, and brain.
Hunter syndrome almost exclusively affects boys and occurs in about 1 in 100,000 male births. Previously, the only available treatment was Elaprase, a drug costing roughly £300,000 per patient per year. While it slows physical symptoms, it cannot cross the blood-brain barrier and therefore does not address cognitive decline.
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