In development: Guidance, quality standards and advice
Showing 1 to 6 of 6
| Title | Type | Expected publication date |
|---|---|---|
| Arimoclomol for treating Niemann-Pick disease Type C [ID1312] | Highly specialised technology | TBC |
| Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (MA review of HST12) [ID6145] | Highly specialised technology | TBC |
| Doxecitine–doxribtimine for treating thymidine kinase 2 deficiency in people of any age [ID6484] | Highly specialised technology | |
| Pegzilarginase for treating arginase-1 deficiency [ID4029] | Highly specialised technology | |
| Tofersen for treating amyotrophic lateral sclerosis caused by SOD1 gene mutations [ID3767] | Highly specialised technology | TBC |
| VTS-270 for treating Niemann-Pick type C1 [ID1267] | Highly specialised technology | TBC |