First gene therapy for rare genetic neurodegenerative disorder in children

Gene therapy

NICE has published a draft highly specialised technologies guidance, recommending gene therapy atidarsagene autotemcel for some children with the rare, life-limiting inherited neurodegenerative disorder metachromatic leukodystrophy (MLD). The draft guidance looks at atidarsagene autotemcel in children with late infantile or early juvenile forms of MLD.

Clinical evidence suggests that atidarsagene autotemcel, the first ever treatment for MLD, improves motor and cognitive function in the short term and could correct the enzyme deficiency caused by the disease.

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