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Showing 1 to 7 of 7 results for mucopolysaccharidosis

  1. Elosulfase alfa for treating mucopolysaccharidosis type 4A (HST19)

    Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type 4A in people of all ages.

    Highly specialised technologies guidance
    Published
    20 April 2022
    View recommendations

    Sections for HST19

  2. UX111 for treating mucopolysaccharidosis type IIIA [ID6540]

    Awaiting development Reference number: GID-TA11701 Expected publication date: TBC

    Technology appraisal guidance
    Awaiting development

  3. Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

    Discontinued Reference number: GID-HST10019

    Highly specialised technologies guidance
    Discontinued

  4. Elosulfase alfa for treating mucopolysaccharidosis type IVa (HST2)

    This guidance has been updated and replaced by NICE’s highly specialised technologies guidance on elosulfase alfa for treating mucopolysaccharidosis type 4A (HST19).

    Withdrawn guidance

  5. Sebelipase alfa for treating Wolman disease (HST30)

    Evidence-based recommendations on sebelipase alfa (Kanuma) for long-term enzyme replacement therapy in Wolman disease (rapidly progressive lysosomal acid lipase deficiency) in people aged 2 years and under when treatment starts.

    Highly specialised technologies guidance
    Published
    10 January 2024
    View recommendations

    Sections for HST30

  6. Rehabilitation for chronic neurological disorders including acquired brain injury (NG252)

    This guideline covers rehabilitation in all settings for children, young people and adults with a chronic neurological disorder, neurological impairment or disabling neurological symptoms due to acquired brain injury, acquired spinal cord injury, acquired peripheral nerve disorder, functional neurological disorder or progressive neurological disease.

    NICE guideline
    Published
    15 October 2025

    Sections for NG252

  7. NICE real-world evidence framework (ECD9)

    This document describes a real-world evidence framework that aims to improve the quality of real-world evidence informing our guidance. The framework does not set minimum standards for the acceptability of evidence. The framework is mainly targeted at those developing evidence to inform NICE guidance. It is also relevant to patients, those collecting data, and reviewers of evidence