Products - Metabolic conditions | Topic

Showing 16 products

Date | Title

Product type: GuidanceRemove ‘Product type: Guidance’ filter
New and updated: New in the last 6 monthsRemove ‘New and updated: New in the last 6 months’ filter

Somapacitan for treating growth hormone deficiency in people 3 to 17 years (TA1066)
Product type:
Guidance
Programme:
Technology appraisal guidance
Published:
3 June 2025
Sodium zirconium cyclosilicate for treating hyperkalaemia (partial review of TA599) [ID6439]
Status:
In development
Programme:
Technology appraisal guidance
Expected publication date:
17 December 2025
Vutrisiran for treating transthyretin-related amyloidosis cardiomyopathy [ID6470]
Status:
In development
Programme:
Technology appraisal guidance
Expected publication date:
28 January 2026
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
CAEL-101 with standard care for untreated amyloid light chain amyloidosis [ID6210]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Pegzilarginase for treating arginase-1 deficiency [ID4029]
Status:
In development
Programme:
Highly specialised technologies guidance
Expected publication date:
TBC
Leriglitazone for treating X-linked andrenoleukodystrophy [TSID9996]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
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
Triheptanoin for treating long-chain fatty acid oxidation disorders [ID3891]
Status:
In development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Eplontersen for treating transthyretin-related amyloidosis cardiomyopathy [TSID12015]
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
Govorestat for treating classic galactosemia in people 2 to 65 years [TSID12071]
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
Elamipretide for treating Barth syndrome in people of any age [ID6545]
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

Metabolic conditions Products

Filter

Product type
1 selected

Guidance programme

Status

New and updated
1 selected

Showing 16 products

Somapacitan for treating growth hormone deficiency in people 3 to 17 years (TA1066)

Sodium zirconium cyclosilicate for treating hyperkalaemia (partial review of TA599) [ID6439]

Vutrisiran for treating transthyretin-related amyloidosis cardiomyopathy [ID6470]

Dersimelagon for treating erythropoietic protoporphyria and X-linked protoporphyria in people 12 years and over [ID6160]

CAEL-101 with standard care for untreated amyloid light chain amyloidosis [ID6210]

Pegzilarginase for treating arginase-1 deficiency [ID4029]

Leriglitazone for treating X-linked andrenoleukodystrophy [TSID9996]

Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease in people 12 to 17 years [TSID11887]

Triheptanoin for treating long-chain fatty acid oxidation disorders [ID3891]

Eplontersen for treating transthyretin-related amyloidosis cardiomyopathy [TSID12015]

Doxecitine–doxribtimine for treating thymidine kinase 2 deficiency in people of any age ID6484

Doxecitine–doxribtimine for treating thymidine kinase 2 deficiency in people of any age who had symptoms before 13 years [TSID12046]

Govorestat for treating classic galactosemia in people 2 to 65 years [TSID12071]

UX111 for treating mucopolysaccharidosis type IIIA [ID6540]

Elamipretide for treating Barth syndrome in people of any age [ID6545]

Veverimer for treating metabolic acidosis in chronic kidney disease [ID3832]

Metabolic conditions Products

Filter

Product type1 selected

Guidance programme

Status

New and updated1 selected

Showing 16 products

Product type
1 selected

New and updated
1 selected