Pegzilarginase for treating arginase-1 deficiency in people 2 years and over (HST35)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 4 March 2026
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (HST34)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 18 February 2026
Burosumab for treating X-linked hypophosphataemia in children and young people (HST8)Product type:GuidanceProgramme:Highly specialised technologies guidanceLast updated: 8 April 2025Published: 10 October 2018
Asfotase alfa for treating paediatric-onset hypophosphatasia (HST23)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 1 March 2023
Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over (HST20)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 5 May 2022
Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 28 March 2022