Published: Guidance, quality standards and advice
Showing 1 to 10 of 29
Title | Reference number | Published | Last updated |
---|---|---|---|
HTE temp withdrawn | HST120 | ||
Setmelanotide for treating obesity and hyperphagia in Bardet-Biedl syndrome | HST31 | ||
Sebelipase alfa for treating Wolman disease | HST30 | ||
Velmanase alfa for treating alpha-mannosidosis | HST29 | ||
Birch bark extract for treating epidermolysis bullosa | HST28 | ||
Afamelanotide for treating erythropoietic protoporphyria | HST27 | ||
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency | HST26 | ||
Lumasiran for treating primary hyperoxaluria type 1 | HST25 | ||
Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy | HST24 | ||
Onasemnogene abeparvovec for treating spinal muscular atrophy | HST15 |