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Guidance programme

Showing 1 to 3 of 3 results for atidarsagene

  1. Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)

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

    Highly specialised technologies guidance
    Published
    28 March 2022
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    Sections for HST18

  2. Pegzilarginase for treating arginase-1 deficiency in people 2 years and over (HST35)

    Evidence-based recommendations on pegzilarginase (Loargys) for treating arginase-1 deficiency in people 2 years and over.

    Highly specialised technologies guidance
    Published
    4 March 2026

    Sections for HST35

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 First gene therapy for rare genetic neurodegenerative disorder in children, recommended in NICE draft guidance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

    News
    Last updated
    18 January 2024