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Guidance programme

Showing 1 to 2 of 2 results for lysosome

  1. Migalastat for treating Fabry disease (HST4)

    Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16.

    Highly specialised technologies guidance
    Published
    22 February 2017
    View recommendations

    Sections for HST4

  2. Olipudase alfa for treating acid sphingomyelinase deficiency (Niemann–Pick disease) type AB and type B (HST32)

    Evidence-based recommendations on olipudase alfa (Xenpozyme) for treating acid sphingomyelinase deficiency (Niemann–Pick disease) in people with type AB or type B.

    Highly specialised technologies guidance
    Published
    2 April 2025
    View recommendations

    Sections for HST32