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11-year-old girl is first UK patient to have gene therapy to halt sight loss
['Storm Newton', 'Press Association Health Reporter']
Irish News
Catherine L’Estrange was diagnosed Bardet-Biedl syndrome (BBS) when she was just a babyAn 11-year-old girl has become the first UK patient to have innovative gene therapy for a rare condition that robs children of their sight.
The gene therapy, developed by biotechnology company MeiraGTx, was given to Catherine as part of an hour-long procedure at St Helier Hospital in March.
The treatment is designed specifically for patients with a BBS10 gene mutation, which is one of the most common among BBS patients.
After having the therapy, patients are seen in clinic and have various sight tests, such as reading from a chart and identifying different shades of colours.
Catherine L’Estrange, 11, from London has become the first UK patient, and second in the world, to have a new gene therapy for a rare condition she was born with.