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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
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
Atidarsagene autotemcel for treating metachromatic leukodystrophy
HST18
28 March 2022
28 March 2022
Birch bark extract for treating epidermolysis bullosa
HST28
20 September 2023
20 September 2023
Burosumab for treating X-linked hypophosphataemia in children and young people
HST8
10 October 2018
10 October 2018
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2
HST12
27 November 2019
27 November 2019
Eculizumab for treating atypical haemolytic uraemic syndrome
HST1
28 January 2015
28 January 2015
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency
HST26
19 April 2023
19 April 2023
Eliglustat for treating type 1 Gaucher disease
HST5
28 June 2017
28 June 2017
Elosulfase alfa for treating mucopolysaccharidosis type 4A
HST19
20 April 2022
20 April 2022
Givosiran for treating acute hepatic porphyria
HST16
24 November 2021
24 November 2021
Inotersen for treating hereditary transthyretin amyloidosis
HST9
22 May 2019
22 May 2019
Lumasiran for treating primary hyperoxaluria type 1
HST25
19 April 2023
19 April 2023
Metreleptin for treating lipodystrophy
HST14
24 February 2021
24 February 2021
Migalastat for treating Fabry disease
HST4
22 February 2017
22 February 2017
Odevixibat for treating progressive familial intrahepatic cholestasis
HST17
22 February 2022
22 February 2022
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
Patisiran for treating hereditary transthyretin amyloidosis
HST10
14 August 2019
14 August 2019
Sebelipase alfa for treating Wolman disease
HST30
10 January 2024
10 January 2024
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
Setmelanotide for treating obesity and hyperphagia in Bardet-Biedl syndrome
HST31
22 May 2024
22 May 2024
Setmelanotide for treating obesity caused by LEPR or POMC deficiency
HST21
6 July 2022
6 July 2022
Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency
HST7
7 February 2018
7 February 2018
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