Published: Guidance, quality standards and advice

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Showing 1 to 10 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

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