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Last updated date between: 1/4/2018 and 28/3/2024
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Published guidance, NICE advice and quality standards
Title
Reference number
Published
Last updated
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
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
Onasemnogene abeparvovec for treating spinal muscular atrophy
HST15
7 July 2021
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
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