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Published guidance, NICE advice and quality standards
Title
Reference number
Published
Last updated
Burosumab for treating X-linked hypophosphataemia in children and young people
HST8
10 October 2018
20 September 2024
Setmelanotide for treating obesity and hyperphagia in Bardet-Biedl syndrome
HST31
22 May 2024
22 May 2024
Sebelipase alfa for treating Wolman disease
HST30
10 January 2024
10 January 2024
Velmanase alfa for treating alpha-mannosidosis
HST29
13 December 2023
13 December 2023
Birch bark extract for treating epidermolysis bullosa
HST28
20 September 2023
20 September 2023
Afamelanotide for treating erythropoietic protoporphyria
HST27
26 July 2023
26 July 2023
Lumasiran for treating primary hyperoxaluria type 1
HST25
19 April 2023
19 April 2023
Onasemnogene abeparvovec for treating spinal muscular atrophy
HST15
7 July 2021
19 April 2023
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency
HST26
19 April 2023
19 April 2023
Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy
HST24
19 April 2023
19 April 2023
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