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Showing 50 products

Date | Title

Afamelanotide for treating erythropoietic protoporphyria (HST27)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
26 July 2023
Asfotase alfa for treating paediatric-onset hypophosphatasia (HST23)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
1 March 2023
Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
28 March 2022
Avalglucosidase alfa for treating Pompe disease (TA821)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
24 August 2022
Burosumab for treating FGF23-related hypophosphataemia in tumour-induced osteomalacia [ID3924 ]
Status:
In development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Burosumab for treating X-linked hypophosphataemia in adults (TA993)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
7 August 2024
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
CAEL-101 with standard care for untreated amyloid light chain amyloidosis [ID6210]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease (TA912)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
15 August 2023
Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease in people 12 to 17 years [TSID11887]
Status:
Topic selection
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Daratumumab in combination for treating newly diagnosed systemic amyloid light-chain amyloidosis (TA959)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
27 March 2024
Dersimelagon for treating erythropoietic protoporphyria and X-linked protoporphyria in people 12 years and over [ID6160]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Doxecitine–doxribtimine for treating thymidine kinase 2 deficiency in people of any age ID6484
Status:
In development
Programme:
Highly specialised technologies guidance
Expected publication date:
TBC
Doxecitine–doxribtimine for treating thymidine kinase 2 deficiency in people of any age who had symptoms before 13 years [TSID12046]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency (HST26)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
19 April 2023
Elamipretide for treating Barth syndrome in people of any age [ID6545]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Eliglustat for treating type 1 Gaucher disease (HST5)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
28 June 2017
Elosulfase alfa for treating mucopolysaccharidosis type 4A (HST19)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
20 April 2022
Eplontersen for treating hereditary transthyretin-related amyloidosis (TA1020)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
27 November 2024
Eplontersen for treating transthyretin-related amyloidosis cardiomyopathy [TSID12015]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Givosiran for treating acute hepatic porphyria (HST16)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
24 November 2021
Govorestat for treating classic galactosemia in people 2 to 65 years [TSID12071]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Inotersen for treating hereditary transthyretin amyloidosis (HST9)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
22 May 2019
Leriglitazone for treating andrenoleukodystrophy [ID3903]
Status:
In development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Leriglitazone for treating X-linked andrenoleukodystrophy [TSID9996]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Migalastat for treating Fabry disease (HST4)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
22 February 2017
Mitapivat for treating pyruvate kinase deficiency (terminated appraisal) (TA867)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
16 February 2023
Mitochondrial disorders in children: Co-enzyme Q10 (ES11)
Product type:
Advice
Programme:
Evidence summary
Published:
28 March 2017
Nitisinone for treating alkaptonuria [ID2691]
Status:
Topic selection
Programme:
Technology appraisal guidance
Expected publication date:
TBC
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
Pegunigalsidase alfa for treating Fabry disease (TA915)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
4 October 2023
Pegzilarginase for treating arginase-1 deficiency [ID4029]
Status:
In development
Programme:
Highly specialised technologies guidance
Expected publication date:
TBC
Preventing recurrent hypomagnesaemia: oral magnesium glycerophosphate (ESUOM4)
Product type:
Advice
Programme:
Evidence summary
Published:
29 January 2013
Prolonged-release diazoxide choline for treating Prader-Willi syndrome [ID1389]
Status:
Topic selection
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Sapropterin for treating hyperphenylalaninaemia in phenylketonuria (TA729)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
22 September 2021
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
Sodium zirconium cyclosilicate for treating hyperkalaemia (TA599)
Product type:
Guidance
Programme:
Technology appraisal guidance
Last updated:
24 January 2022
Published:
4 September 2019
Sodium zirconium cyclosilicate for treating hyperkalaemia (partial review of TA599) [ID6439]
Status:
In development
Programme:
Technology appraisal guidance
Expected publication date:
17 December 2025
Somapacitan for treating growth hormone deficiency in people 3 to 17 years [ID6178]
Status:
In development
Programme:
Technology appraisal guidance
Expected publication date:
29 May 2025
Tafamidis for treating transthyretin amyloidosis with cardiomyopathy (TA984)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
19 June 2024
Triheptanoin for treating long-chain fatty acid oxidation disorders [ID3891]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
UX111 for treating mucopolysaccharidosis type IIIA [ID6540]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Velmanase alfa for treating alpha-mannosidosis (HST29)
Product type:
Guidance
Programme:
Highly specialised technologies guidance
Published:
13 December 2023
Venglustat for treating gangliosidoses in people 2 years and over ID 6358
Status:
In development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Venglustat for treating type 3 Gaucher disease in people aged 12 and over after at least 3 years of enzyme replacement therapy [ID6295]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Veverimer for treating metabolic acidosis in chronic kidney disease [ID3832]
Status:
Topic selection
Programme:
Technology appraisal guidance
Expected publication date:
TBC
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
Vutrisiran for treating hereditary transthyretin-related amyloidosis (TA868)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
15 February 2023
Vutrisiran for treating transthyretin-related amyloidosis cardiomyopathy [ID6470]
Status:
In development
Programme:
Technology appraisal guidance
Expected publication date:
28 January 2026