In development: Guidance, quality standards and advice
Showing 1 to 5 of 5
| Title | Type | Expected publication date |
|---|---|---|
| Arimoclomol for treating Niemann-Pick disease Type C [ID1312] | Highly specialised technology | TBC |
| Doxecitine–doxribtimine for treating thymidine kinase 2 deficiency in people of any age [ID6484] | Highly specialised technology | TBC |
| Pegzilarginase for treating arginase-1 deficiency in people 2 years and over [ID4029] | Highly specialised technology | |
| 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 | TBC |