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
Volanesorsen for treating familial chylomicronaemia syndrome (HST13)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 21 October 2020
Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 28 January 2015
OTL-101 for treating adenosine deaminase deficiency–severe combined immunodeficiency [ID1152]Status:Awaiting developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC