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Published guidance, NICE advice and quality standards
Title
Reference number
Published
Last updated
Afamelanotide for treating erythropoietic protoporphyria
HST27
26 July 2023
26 July 2023
Onasemnogene abeparvovec for treating spinal muscular atrophy
HST15
7 July 2021
19 April 2023
Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy
HST24
19 April 2023
19 April 2023
Lumasiran for treating primary hyperoxaluria type 1
HST25
19 April 2023
19 April 2023
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency
HST26
19 April 2023
19 April 2023
Asfotase alfa for treating paediatric-onset hypophosphatasia
HST23
1 March 2023
1 March 2023
Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene
HST22
22 February 2023
22 February 2023
Setmelanotide for treating obesity caused by LEPR or POMC deficiency
HST21
6 July 2022
6 July 2022
Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over
HST20
5 May 2022
5 May 2022
Elosulfase alfa for treating mucopolysaccharidosis type 4A
HST19
20 April 2022
20 April 2022
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