Published: Guidance, quality standards and advice

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Showing 21 to 30 of 35

TitleReference numberPublishedLast updated
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
Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiencyHST7
Eliglustat for treating type 1 Gaucher diseaseHST5
Managing medicines for adults receiving social care in the communityNG67
Patient group directionsMPG2

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