Dersimelagon for treating erythropoietic protoporphyria and X-linked protoporphyria in people 12 years and over [ID6160]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
Pariglasgene brecaparvovec for treating glycogen storage disease type 1a in people 8 years and over [TSID11865]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 [ID6759]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 [ID6484]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Efzimfotase alfa for treating hypophosphatasia [ID6633]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Sepiapterin for treating hyperphenylalaninaemia in phenylketonuria in people of any age [ID6750]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC