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Guidance programme

Showing 21 to 28 of 28

Published guidance, NICE advice and quality standards
TitleReference numberPublishedLast updated
Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutationsHST11
Patisiran for treating hereditary transthyretin amyloidosisHST10
Inotersen for treating hereditary transthyretin amyloidosisHST9
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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