Summary
A three-year-old boy named Oliver Chu from California is the first person with Hunter syndrome to receive a new gene therapy in Manchester, UK. This pioneering treatment aims to address the genetic cause of the disease, and a year after starting, Oliver is showing normal development.
Key Facts
- Oliver Chu, aged three, has a rare genetic condition called Hunter syndrome.
- Hunter syndrome can lead to severe damage in the body and brain, often resulting in early death.
- Oliver was the first person to receive a new gene therapy for this condition.
- The procedure took place at Royal Manchester Children's Hospital in the UK.
- Gene therapy involved altering Oliver's cells to produce a missing enzyme.
- Hunter syndrome affects mostly boys and is very rare, occurring in about 1 in 100,000 male births.
- Previously, available treatments like Elaprase could only slow physical symptoms but not cognitive ones.