Velmanase alfa for treating alpha-mannosidosis (HST29)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 13 December 2023
Afamelanotide for treating erythropoietic protoporphyria (HST27)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 26 July 2023
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency (HST26)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 19 April 2023
Asfotase alfa for treating paediatric-onset hypophosphatasia (HST23)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 1 March 2023
Elosulfase alfa for treating mucopolysaccharidosis type 4A (HST19)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 20 April 2022
Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 28 March 2022
Givosiran for treating acute hepatic porphyria (HST16)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 24 November 2021
Patisiran for treating hereditary transthyretin amyloidosis (HST10)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 14 August 2019
Inotersen for treating hereditary transthyretin amyloidosis (HST9)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 22 May 2019
Eliglustat for treating type 1 Gaucher disease (HST5)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 28 June 2017
Migalastat for treating Fabry disease (HST4)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 22 February 2017
Pegzilarginase for treating arginase-1 deficiency [ID4029]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC