The treatment, Libmeldy, was approved by the NHS last year for the genetic condition and works by correcting the cause of MLD, inserting functional copies of a faulty gene into the patient’s own stem cells. The stem cells come from the patient’s bone marrow or blood, and then feed back into the body, carrying the functional genetic material.
Teddi had her stem cells removed and the faulty genes replaced between June and October last year and is now said to be "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... she has the opportunity to lead a long and hopefully normal life."
Professor Rob Wynn, director of paediatric bone marrow transplants at Royal Manchester commented, "Through the years, colleagues and I have looked after a range of patients with rare but severe conditions, where treatment has been limited... It is wonderful to be involved in this breakthrough moment and deliver a gene therapy which will transform outcomes for patients with MLD."
Despite MLD being very rare, with only 5 children in England being born with the condition last year, Teddi's older sister, Nala, also suffers but the treatment has come too late as she has progressed too far in her condition for it to be effective.
Ally, Nala and Teddi's mother, commented, "In April last year, our world was turned upside down when not one, but both of our daughters were diagnosed with MLD... Being told our first daughter, Nala, wasn’t eligible for any treatment, would continue to lose all functions and die extremely young was the most heartbreaking and hardest thing to come to terms with."