Asfotase alfa for treating paediatric-onset hypophosphatasia (HST23)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 1 March 2023
Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 28 March 2022
Burosumab for treating X-linked hypophosphataemia in children and young people (HST8)Product type:GuidanceProgramme:Highly specialised technologies guidanceLast updated: 20 September 2024Published: 10 October 2018
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (HST12)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 27 November 2019
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (review of HST12) [ID6145]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC
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
VTS-270 for treating Niemann-Pick type C1 [ID1267]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC