Guidance, NICE advice and quality standards In development

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Showing 1 to 10 of 10

TitleTypeExpected publication date
Arimoclomol for treating Niemann-Pick disease Type C [ID1312]Highly specialised technologyTBC
Fosdenopterin for treating molybdenum cofactor deficiency type A ID6264Highly specialised technology
Human alpha1-proteinase inhibitor for treating emphysema ID856Highly 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 as an add-on treatment for arginase-1 deficiency [ID4029]Highly specialised technologyTBC
Sebelipase alfa for treating lysosomal acid lipase deficiency [ID737]Highly specialised technologyTBC
Sebelipase alfa for treating Wolman disease [ID3995]Highly specialised technology
Velmanase alfa for treating alpha-mannosidosis [ID800]Highly specialised technology
VTS-270 for treating Niemann-Pick type C1 [ID1267]Highly specialised technologyTBC

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