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Guidance programme (1 selected)

Guidance programme

Showing 1 to 10 of 27

Published guidance, NICE advice and quality standards
TitleReference numberPublishedLast updated
Sebelipase alfa for treating Wolman diseaseHST30
Velmanase alfa for treating alpha-mannosidosisHST29
Birch bark extract for treating epidermolysis bullosaHST28
Afamelanotide for treating erythropoietic protoporphyriaHST27
Lumasiran for treating primary hyperoxaluria type 1HST25
Onasemnogene abeparvovec for treating spinal muscular atrophyHST15
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiencyHST26
Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophyHST24
Asfotase alfa for treating paediatric-onset hypophosphatasiaHST23
Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin geneHST22

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