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Guidance programme

Showing 21 to 25 of 25

Published guidance, NICE advice and quality standards
TitleReference numberPublishedLast updated
Burosumab for treating X-linked hypophosphataemia in children and young peopleHST8
Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiencyHST7
Eliglustat for treating type 1 Gaucher diseaseHST5
Migalastat for treating Fabry diseaseHST4
Eculizumab for treating atypical haemolytic uraemic syndromeHST1

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