Guidance, NICE advice and quality standards In development
Showing 1 to 8 of 8
Title | Type | Expected publication date |
---|---|---|
Arimoclomol for treating Niemann-Pick disease Type C [ID1312] | Highly specialised technology | TBC |
Fosdenopterin for treating molybdenum cofactor deficiency type A [ID6264] | Highly specialised technology | |
Leniolisib for activated phosphoinositide 3-kinase delta syndrome in people 12 years and over [ID6130] | Highly specialised technology | |
Olipudase alfa for treating Niemann-Pick disease types A and B [ID3913 ] | Highly specialised technology | TBC |
Palovarotene for preventing heterotopic ossification associated with fibrodysplasia ossificans progressiva [ID3739] | Highly specialised technology | TBC |
Palovarotene for treating flare ups of heterotopic ossification associated with fibrodysplasia ossificans progressiva [ID2721] | Highly specialised technology | TBC |
Pegzilarginase for treating arginase-1 deficiency [ID4029] | Highly specialised technology | |
VTS-270 for treating Niemann-Pick type C1 [ID1267] | Highly specialised technology | TBC |