Eplontersen for treating hereditary transthyretin-related amyloidosis (TA1020)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 27 November 2024
Burosumab for treating X-linked hypophosphataemia in adults (TA993)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 7 August 2024
Tafamidis for treating transthyretin amyloidosis with cardiomyopathy (TA984)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 19 June 2024
Daratumumab in combination for treating newly diagnosed systemic amyloid light-chain amyloidosis (TA959)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 27 March 2024
Velmanase alfa for treating alpha-mannosidosis (HST29)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 13 December 2023
Pegunigalsidase alfa for treating Fabry disease (TA915)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 4 October 2023
Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease (TA912)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 15 August 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
Vutrisiran for treating hereditary transthyretin-related amyloidosis (TA868)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 15 February 2023
Somatrogon for treating growth disturbance in children and young people aged 3 years and over (TA863)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 1 February 2023
Slow-release potassium bicarbonate–potassium citrate for treating distal renal tubular acidosis (terminated appraisal) (TA838)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 2 November 2022
Avalglucosidase alfa for treating Pompe disease (TA821)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 24 August 2022
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
Sapropterin for treating hyperphenylalaninaemia in phenylketonuria (TA729)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 22 September 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
Vitamin D: supplement use in specific population groups (PH56)Product type:GuidanceProgramme:Public health guidelineLast updated: 30 August 2017Published: 26 November 2014
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
Human growth hormone (somatropin) in adults with growth hormone deficiency (TA64)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 27 August 2003
Macimorelin for diagnosing growth hormone deficiency (MIB320)Product type:AdviceProgramme:Medtech innovation briefingPublished: 28 March 2023