In development: Guidance, quality standards and advice

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TitleTypeExpected publication date
Arimoclomol for treating Niemann-Pick disease Type C [ID1312]Highly specialised technologyTBC
Doxecitine–doxribtimine for treating thymidine kinase 2 deficiency in people of any age [ID6484]Highly specialised technologyTBC
Setmelanotide for treating obesity and hyperphagia in Bardet-Biedl syndrome (review of HST31) [ID6598]Highly specialised technology
Tofersen for treating amyotrophic lateral sclerosis caused by SOD1 gene mutations [ID3767]Highly specialised technology

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