Dersimelagon for treating erythropoietic protoporphyria and X-linked protoporphyria in people 12 years and over [TSID10392]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
CAEL-101 with standard care for untreated amyloid light chain amyloidosis [ID6210]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected 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 developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Leriglitazone for treating X-linked andrenoleukodystrophy [TSID9996]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Triheptanoin for treating long-chain fatty acid oxidation disorders [ID3891]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Eplontersen for treating transthyretin-related amyloidosis cardiomyopathy [TSID12015]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected 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 developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Govorestat for treating classic galactosemia in people 2 to 65 years [TSID12071]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
UX111 for treating mucopolysaccharidosis type IIIA [ID6540]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Elamipretide for treating Barth syndrome in people of any age [ID6545]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC