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Royal Manchester Russia hospital patient travelers Royal Manchester Russia

Little girl travels to Manchester from Russia to become first child in the world to receive pioneering gene therapy

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manchestereveningnews.co.ukmanchestereveningnews.co.uk:

Don't miss a thing by getting the day's biggest stories sent direct to your inbox A little girl with a rare genetic disorder has travelled from Russia to take part in a pioneering trial at the Royal Manchester Children's Hospital.

Two-year-old Stefaniia has a metabolic disease known as GM1 gangliosidosis - a genetic condition affecting the body’s chemistry.

GM1 results in severe and life-threatening damage to the brain and spinal cord, and can lead to early death. READ MORE: All adults to be able to book Covid-19 vaccine ‘by the end of the week’ There is currently no treatment available for this devastating disease.

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