Published Guidance, NICE advice and quality standards

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Showing 1 to 10 of 25

TitleReference numberPublishedLast updated
Birch bark extract for treating epidermolysis bullosaHST28
Afamelanotide for treating erythropoietic protoporphyriaHST27
Onasemnogene abeparvovec for treating spinal muscular atrophyHST15
Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophyHST24
Lumasiran for treating primary hyperoxaluria type 1HST25
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiencyHST26
Asfotase alfa for treating paediatric-onset hypophosphatasiaHST23
Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin geneHST22
Setmelanotide for treating obesity caused by LEPR or POMC deficiencyHST21
Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and overHST20

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