A child’s hand on a hospital bed with intravenous drip attached
A child's hand on a hospital bed with intravenous drip attached - A girl born with a rare and fatal genetic condition has been potentially cured of her disease thanks to a revolutionary gene therapy developed by UCL spinout Orchard Therapeutics (Orchard). Nineteen-month-old Teddi Shaw, from Northumberland, was diagnosed with metachromatic leukodystrophy (MLD), which causes severe damage to the nervous system and leads to children being unable to move, talk, swallow, eat and see. There are no treatment options for children with MLD and children are offered only supportive care and invariably die young. But after becoming the first person to receive treatment on the NHS with pioneering drug Libmeldy, Teddi may now look forward to a disease-free life. Orchard's development programmes focus on restoring normal gene function in severe devastating genetic disorders including neurometabolic diseases such as MLD. The spinout was formed as a result of more than two decades of gene therapy research and clinical work by UCL Professors Bobby Gaspar and Adrian Thrasher (both UCL Great Ormond Street Institute of Child Health). Their ultimate aim was to bring potentially curative gene therapy medicines to patients with severe devastating diseases.
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