This is the first Lenmeldy gene therapy for children with metachromatic leukodystrophy.

Metachromatic leukodystrophy is a rare hereditary disease that leads to irreversible damage to the brain, other organs and the nervous system. It is manifested by loss of motor and cognitive functions and can cause early death – many children with this disease do not live to the age of ten.

Lenmeldy from the British company Orchard Therapeutics has become the first registered gene drug for the treatment of this disease - previously it consisted of supportive therapy and symptom control.

37 children participated in the FDA clinical trials. The only time they were given a single dose of a drug made from their own hematopoietic (blood) stem cells, which were genetically modified. The study showed that the administration of the drug significantly reduced the risk of severe motor impairment or death compared with children who did not receive Lenmeldy. And according to the FDA, 71% of children receiving therapy could walk unaided by the age of five, and 85% had normal IQ and speech scores.