Published: Guidance, quality standards and advice

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Showing 21 to 28 of 28

TitleReference numberPublishedLast updated
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2HST12
Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutationsHST11
Patisiran for treating hereditary transthyretin amyloidosisHST10
Inotersen for treating hereditary transthyretin amyloidosisHST9
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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