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Toddler becomes first child to receive gene therapy for fatal disorder on the NHS

BMJ 2023; 380 doi: (Published 15 February 2023) Cite this as: BMJ 2023;380:p370
  1. Elisabeth Mahase
  1. The BMJ

A 19 month old child named Teddi has received life saving gene therapy treatment for metachromatic leukodystrophy (MLD)—a disorder that causes severe damage to the nervous system and organs—in an NHS first.

The one time treatment, Libmeldy (atidarsagene autotemcel), works by taking the patient’s stem cells, replacing the faulty gene that causes MLD, and then reinjecting the treated cells back into the patient.

It is listed as the “most expensive drug” in the world at £2.8m; NHS England is, however, understood to have negotiated a “significant confidential discount” last year.

According to the National Institute for Health and Care Excellence, the clinical evidence for Libmeldy suggests it “improves mobility and cognitive function and could correct the enzyme deficiency caused by the …

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