Published: Guidance, quality standards and advice

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Guidance programme 1 selected

Guidance programme

Showing 11 to 20 of 30

TitleReference numberPublishedLast updated
Elosulfase alfa for treating mucopolysaccharidosis type 4AHST19
Givosiran for treating acute hepatic porphyriaHST16
Inotersen for treating hereditary transthyretin amyloidosisHST9
Leniolisib for treating activated phosphoinositide 3-kinase delta syndrome in people 12 years and overHST33
Lumasiran for treating primary hyperoxaluria type 1HST25
Metreleptin for treating lipodystrophyHST14
Migalastat for treating Fabry diseaseHST4
Odevixibat for treating progressive familial intrahepatic cholestasisHST17
Olipudase alfa for treating acid sphingomyelinase deficiency (Niemann–Pick disease) type AB and type BHST32
Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophyHST24

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