World-Frist Gene Therapy Transforms Life of Boy with HunterS Disease
Stockholm, Sweden – A young boy named Oliver is thriving after becoming the first person in the world to receive gene therapy for Hunter’s disease, a rare and often fatal genetic disorder. One year into treatment, Oliver is exhibiting normal development, producing a missing enzyme crucial for bodily function, and experiencing a dramatic improvement in his overall health.
Hunter’s disease, or Mucopolysaccharidosis type II, primarily affects boys, impacting airways, the heart, joints, bones, liver, hearing, and cognitive development. Affecting approximately one in 100,000 boys globally, and an estimated 10-15 people in Sweden, the disease currently has no cure, with existing treatments focused solely on managing symptoms. This groundbreaking gene therapy offers a potential path toward lasting correction of the underlying genetic defect.
Oliver’s father, Ricky, describes the transformation as remarkable. “he is like a completely different child. He runs around everywhere and talks non-stop,” he said. Prior to the treatment, Oliver suffered from the debilitating effects of the disease. Now, his body and brain are healthy, offering hope for a future previously unimaginable.
The treatment, a collaborative effort between a hospital and a university in Sweden, involves delivering functional genes to correct the deficiency causing Hunter’s disease. All five boys included in the initial study will be monitored for at least two years.If the results continue to be positive, researchers hope to secure approval for wider use of the therapy, possibly offering a life-altering treatment to children worldwide.
“Every time we talk about it, I want to cry as it’s just so amazing,” Oliver’s mother, Jingru, told the BBC.
About Hunter’s Disease:
The disease is linked to the X chromosome, explaining its near-exclusive occurrence in boys. The severe form of Hunter’s disease frequently enough leads to impaired development and a shortened lifespan. Current treatment strategies aim to alleviate symptoms,prevent medical complications,and compensate for functional impairments,but do not address the root cause of the illness. (Source: National Board of Health and Welfare)
