Published: Guidance, quality standards and advice

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Showing 1 to 28 of 28

TitleReference numberPublishedLast updated
Burosumab for treating X-linked hypophosphataemia in children and young peopleHST8
Setmelanotide for treating obesity and hyperphagia in Bardet-Biedl syndromeHST31
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
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
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
Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiencyHST7
Eliglustat for treating type 1 Gaucher diseaseHST5
Migalastat for treating Fabry diseaseHST4
Eculizumab for treating atypical haemolytic uraemic syndromeHST1

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