Published Guidance, NICE advice and quality standards

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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 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
Velmanase alfa for treating alpha-mannosidosis	HST29	13 December 2023	13 December 2023
Volanesorsen for treating familial chylomicronaemia syndrome	HST13	21 October 2020	21 October 2020
Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations	HST11	9 October 2019	9 October 2019

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