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
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (review of HST12) [ID6145]Highly specialised technologyTBC
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 technologyTBC
Olipudase alfa for treating Niemann-Pick disease types A and B [ID3913 ]Highly specialised technologyTBC
Palovarotene for preventing heterotopic ossification associated with fibrodysplasia ossificans progressiva [ID3739]Highly specialised technologyTBC
Palovarotene for treating flare ups of heterotopic ossification associated with fibrodysplasia ossificans progressiva [ID2721]Highly specialised technologyTBC
Pegzilarginase for treating arginase-1 deficiency [ID4029]Highly specialised technologyTBC
Tofersen for treating amyotrophic lateral sclerosis caused by SOD1 gene mutations [ID3767]Highly specialised technologyTBC
VTS-270 for treating Niemann-Pick type C1 [ID1267]Highly specialised technologyTBC

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