Sebelipase alfa for treating Wolman disease (HST30)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 10 January 2024
Metreleptin for treating lipodystrophy (HST14)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 24 February 2021
VTS-270 for treating Niemann-Pick type C1 [ID1267]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC
Arimoclomol for treating Niemann-Pick disease Type C [ID1312]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC
Olipudase alfa for treating Niemann-Pick disease types A and B [ID3913 ]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC