Royal Manchester Children’s Hospital patient ‘doing well’ after receiving world-first gene therapy treatment for life-limiting genetic condition

Press/Media: Research

Description

A child with a life-limiting condition, who was the first person in the world to receive a pioneering gene therapy, is ‘doing well’ almost 10 months on from treatment.

The Royal Manchester Children’s Hospital (RMCH) patient was two-years-old when he received the new investigational treatment for the genetic condition, Mucopolysaccharidosis IIIA (MPSIIIA), in January 2019.

Period5 Nov 2019

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