Published guidance, NICE advice and quality standards | Guidance

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Published guidance, NICE advice and quality standards
Title	Reference number	Published	Last updated
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
Atidarsagene autotemcel for treating metachromatic leukodystrophy	HST18	28 March 2022	28 March 2022
Odevixibat for treating progressive familial intrahepatic cholestasis	HST17	22 February 2022	22 February 2022
Givosiran for treating acute hepatic porphyria	HST16	24 November 2021	24 November 2021
Metreleptin for treating lipodystrophy	HST14	24 February 2021	24 February 2021
Volanesorsen for treating familial chylomicronaemia syndrome	HST13	21 October 2020	21 October 2020