Asfotase alfa for treating paediatric-onset hypophosphatasia (HST23)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 1 March 2023
Fosdenopterin for treating molybdenum cofactor deficiency type A [ID6264]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: 29 January 2025
Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy (HST24)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 19 April 2023
Onasemnogene abeparvovec for treating spinal muscular atrophy (HST15)Product type:GuidanceProgramme:Highly specialised technologies guidanceLast updated: 19 April 2023Published: 7 July 2021
VTS-270 for treating Niemann-Pick type C1 [ID1267]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC