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Getting the latest healthcare news for you
Getting the latest healthcare news for you
Scotland's NHS just approved a one-time gene therapy for metachromatic leukodystrophy (MLD), a rare and fatal childhood neurological disorder. Atidarsagene autotemcel (Libmeldy) works by correcting the genetic defect that causes toxic buildup in the brain and nerves. Long-term data show it dramatically improves survival and motor function — especially when given before symptoms appear.
The Scottish Medicines Consortium (SMC) has approved atidarsagene autotemcel (Libmeldy) for routine use on NHS Scotland to treat metachromatic leukodystrophy (MLD) — a rare, inherited disorder that destroys the nervous system in young children. The one-time gene therapy uses a patient's own stem cells, modified to carry a working copy of the faulty ARSA gene, and reinfused to restore the enzyme that prevents toxic sulfatide buildup in the brain and nerves.
The approval follows a reassessment under the ultra-orphan pathway, supported by compelling long-term clinical evidence and patient advocacy testimony. Treatment is indicated for presymptomatic children with late-infantile or early-juvenile MLD, and for early-juvenile patients who can still walk independently.
By the Numbers:
Why it matters: MLD has historically had no disease-modifying treatment — only supportive care. This approval offers children a genuine chance at a near-normal life, but early diagnosis is critical. Experts are calling for newborn screening programs to identify presymptomatic cases before the window for effective treatment closes.
