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Guidance programme

Showing 1 to 2 of 2 results for atidarsagene autotemcel

  1. Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)

    Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children.

  2. First gene therapy for rare genetic neurodegenerative disorder in children, recommended in NICE draft guidance

    Children with a rare, fatal, genetic disorder will be able to benefit from a new one-off treatment under new NICE guidance.