Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy (HST24)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 19 April 2023
Maternal and child nutritionStatus:In developmentProgramme:NICE guidelineExpected publication date: 10 July 2024
VTS-270 for treating Niemann-Pick type C1 [ID1267]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC
Fosdenopterin for treating molybdenum cofactor deficiency type A ID6264Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC