Products - Metabolic conditions | Topic

Showing 11 products

Date | Title

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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
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
Leriglitazone for treating X-linked andrenoleukodystrophy [TSID9996]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Triheptanoin for treating long-chain fatty acid oxidation disorders [ID3891]
Status:
Awaiting 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 who had symptoms before 13 years [TSID12046]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Rebisufligene etisparvovec 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
Efzimfotase alfa for treating hypophosphatasia [ID6633]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC
Sepiapterin for treating hyperphenylalaninaemia in phenylketonuria in people of any age [ID6750]
Status:
Awaiting development
Programme:
Technology appraisal guidance
Expected publication date:
TBC

Metabolic conditions Products

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Product type

Guidance programme

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New and updated

Showing 11 products

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]

Venglustat for treating type 3 Gaucher disease in people aged 12 and over after at least 3 years of enzyme replacement therapy [ID6295]

Leriglitazone for treating X-linked andrenoleukodystrophy [TSID9996]

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 who had symptoms before 13 years [TSID12046]

Rebisufligene etisparvovec for treating mucopolysaccharidosis type IIIA [ID6540]

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

Efzimfotase alfa for treating hypophosphatasia [ID6633]

Sepiapterin for treating hyperphenylalaninaemia in phenylketonuria in people of any age [ID6750]

Metabolic conditions Products

Filter

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Guidance programme

Status1 selected

New and updated

Showing 11 products

Status
1 selected