Published Guidance, NICE advice and quality standards

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Guidance programme

Showing 21 to 28 of 28

TitleReference numberPublishedLast updated
Sebelipase alfa for treating Wolman diseaseHST30
Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and overHST20
Setmelanotide for treating obesity and hyperphagia in Bardet-Biedl syndromeHST31
Setmelanotide for treating obesity caused by LEPR or POMC deficiencyHST21
Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiencyHST7
Velmanase alfa for treating alpha-mannosidosisHST29
Volanesorsen for treating familial chylomicronaemia syndromeHST13
Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutationsHST11

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