A young girl suffering from a rare and fatal disease became the first patient in the United Kingdom to receive a revolutionary gene therapy, and today, she has the chance to live a normal life, giving hope to families having to deal with the deadly genetic disorder.
“This is a huge moment of hope for parents and their babies who are born with this devastating inherited disorder, that can now be treated with a single round of revolutionary treatment at a specialist centre on the NHS,” states NHS chief executive Amanda Pritchard.
Teddi Shaw, a 19-month-old girl from Shilbottle, Northumberland, is afflicted with deadly metachromatic leukodystrophy (MLD) which causes serious damage to the nervous system and organs. Around one in five children is born with MLD each year in England. The toddler was administered Libmeldy, a drug that corrects the genetic cause of MLD by inserting functional copies of a faulty gene into her own stem cells. Listed at $3.36 million, the drug was sold at a discounted price thanks to an agreement between NHS England and the biotech company Orchard Therapeutics. Teddi received a one-off intravenous infusion last year and already shows no signs of the fatal disorder she was born with. “Teddi is doing absolutely brilliant. She is walking, running, a chatterbox – absolutely no signs so far of MLD. She is an absolute character and has everyone around her laughing all the time,” rejoices the little girl’s mother.