UK-based Orchard Therapeutics has received FDA approval for gene therapy "Lenmeldy" to treat children suffering from metachromatic leukodystrophy (MLD), a rare genetic disease. The therapy's price has not been disclosed.
Lenmeldy is a one-time therapy for children experiencing certain stages of MLD and uses the patient's own hematopoietic stem cells. The FDA reported that Lenmeldy substantially reduced the risk of severe motor impairment or death compared to untreated children. In December 2020, the therapy was approved and marketed in the European Union under the brand name "Libmeldy."
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