Published Guidance, NICE advice and quality standards

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Guidance programme

Showing 11 to 20 of 25

TitleReference numberPublishedLast updated
Elosulfase alfa for treating mucopolysaccharidosis type 4AHST19
Atidarsagene autotemcel for treating metachromatic leukodystrophyHST18
Odevixibat for treating progressive familial intrahepatic cholestasisHST17
Givosiran for treating acute hepatic porphyriaHST16
Metreleptin for treating lipodystrophyHST14
Volanesorsen for treating familial chylomicronaemia syndromeHST13
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

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