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19-month-old Teddi Shaw was diagnosed with MLD. The condition causes the progressive destruction of the protective covering of nerve fibers in the brain and spinal cord, leading to a range of devastating symptoms including loss of motor skills, blindness, deafness, and ultimately, death.
In a medical breakthrough, she has been cured using gene therapy.
The treatment, called Libmeldy, was administered at Great Ormond Street Hospital in London, and was made possible thanks to a collaboration between the UK’s National Health Service (NHS) and Orchard Therapeutics, a gene therapy company.
Until now, there has been no cure for the disease, with the only available treatment being bone marrow transplantation, which is often not effective.
Replacing the defective gene with a healthy one
The gene therapy treatment works by replacing the defective gene responsible for MLD with a healthy version of the gene. The new gene is delivered to the patient’s body via a harmless virus. After receiving the treatment, the young girl’s condition has significantly improved, with her motor skills and cognitive abilities returning to normal.
The success of the treatment is not only a breakthrough for the young girl and her family but for all those who suffer from MLD. The NHS has announced that it will be offering the treatment to other MLD patients who are eligible, making it the first time a gene therapy has been made available on the NHS for this specific condition.
The chief executive of NHS England, Amanda Pritchard, said to the Guardian.
“This is a huge moment of hope for parents and their babies who are born with this devastating inherited disorder."
The success of the treatment also marks a significant step forward for gene therapy as a whole. Gene therapy has long been hailed as a potential solution for a range of genetic diseases, but until now, there have been few successful treatments. This breakthrough could pave the way for new gene therapies for other rare and deadly conditions.
News tips: Tony Morley
- The Guardian. (2023, February 15). Girl with deadly inherited condition MLD cured by gene therapy. Retrieved from https://www.theguardian.com/society/2023/feb/15/girl-with-deadly-inherited-condition-mld-cured-gene-therapy-libmeldy-nhs