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Boy with rare condition amazes doctors after world-first gene therapy

Boy with rare condition amazes doctors after world-first gene therapy

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.

Source Information