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Showing 28 products

Date | Title

Status: PublishedRemove ‘Status: Published’ filter

Burosumab for treating X-linked hypophosphataemia in children and young people (HST8)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Last updated:
8 April 2025
Published:
10 October 2018
Eplontersen for treating hereditary transthyretin-related amyloidosis (TA1020)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
27 November 2024
Burosumab for treating X-linked hypophosphataemia in adults (TA993)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
7 August 2024
Tafamidis for treating transthyretin amyloidosis with cardiomyopathy (TA984)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
19 June 2024
Daratumumab in combination for treating newly diagnosed systemic amyloid light-chain amyloidosis (TA959)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
27 March 2024
Velmanase alfa for treating alpha-mannosidosis (HST29)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
13 December 2023
Pegunigalsidase alfa for treating Fabry disease (TA915)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
4 October 2023
Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease (TA912)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
15 August 2023
Afamelanotide for treating erythropoietic protoporphyria (HST27)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
26 July 2023
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency (HST26)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
19 April 2023
Asfotase alfa for treating paediatric-onset hypophosphatasia (HST23)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
1 March 2023
Mitapivat for treating pyruvate kinase deficiency (terminated appraisal) (TA867)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
16 February 2023
Vutrisiran for treating hereditary transthyretin-related amyloidosis (TA868)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
15 February 2023
Slow-release potassium bicarbonate–potassium citrate for treating distal renal tubular acidosis (terminated appraisal) (TA838)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
2 November 2022
Avalglucosidase alfa for treating Pompe disease (TA821)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
24 August 2022
Elosulfase alfa for treating mucopolysaccharidosis type 4A (HST19)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
20 April 2022
Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
28 March 2022
Sodium zirconium cyclosilicate for treating hyperkalaemia (TA599)
Product type:
Guidance
Programme:
Technology appraisal guidance
Last updated:
24 January 2022
Published:
4 September 2019
Givosiran for treating acute hepatic porphyria (HST16)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
24 November 2021
Sapropterin for treating hyperphenylalaninaemia in phenylketonuria (TA729)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
22 September 2021
Patiromer for treating hyperkalaemia (TA623)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
13 February 2020
Patisiran for treating hereditary transthyretin amyloidosis (HST10)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
14 August 2019
Inotersen for treating hereditary transthyretin amyloidosis (HST9)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
22 May 2019
Vitamin D: supplement use in specific population groups (PH56)
Product type:
Guidance
Programme:
Public health guideline
Last updated:
30 August 2017
Published:
26 November 2014
Eliglustat for treating type 1 Gaucher disease (HST5)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
28 June 2017
Migalastat for treating Fabry disease (HST4)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
22 February 2017
Mitochondrial disorders in children: Co-enzyme Q10 (ES11)
Product type:
Advice
Programme:
Evidence summary
Published:
28 March 2017
Preventing recurrent hypomagnesaemia: oral magnesium glycerophosphate (ESUOM4)
Product type:
Advice
Programme:
Evidence summary
Published:
29 January 2013