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| Status | Drug name | NICE TAs | Comments |
|---|---|---|---|
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Agalsidase alpha infusion |
NHSE commissioned. |
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Agalsidase beta |
NHSE commissioned |
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Alglucosidase alfa injection |
NHSE commissioned. |
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Asfotase alfa |
NHSE commissioned |
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Betaine anhydrous powder (180g) |
NHSE commissioned. |
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Carglumic acid |
NHSE commissioned |
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Carglumic acid dispersible tablets |
NHSE commissioned. |
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Cipaglucosidase alfa | ||
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Eliglustat (Cerdelga®) |
NHSE commissioned |
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Elosulfase lfa (Vimizim®) |
NHSE commissioned |
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Galsulfase (Naglazyme®) |
NHSE commissioned |
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Glycerol phenylbutyrate |
NHSE commissioned |
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Haem arginate (Normasang) |
NHSE commissioned. |
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Idursulfase concentrate for infusion |
NHSE commissioned. |
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Imiglucerase injection |
NHSE commissioned. |
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L-arginine capsules | ||
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L-arginine infusion (100ml) | ||
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L-arginine oral solution | ||
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L-arginine tablets | ||
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L-carnitine chewable tablet |
NHSE commissioned. |
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L-carnitine injection |
NHSE commissioned. |
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L-carnitine liquid |
NHSE commissioned. |
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L-ornithine L-aspartate 5gm/10ml injection | ||
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Laronidase infusion (Aldurazyme) | ||
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Mercaptamine capsules |
NHSE commissioned. |
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Migalastat (Galafold) | ||
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Miglustat (Zavesca) | ||
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Nitisinone capsules | ||
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Pegunigalsidase alfa (Elfabrio®) | ||
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Penicillamine |
Specialist recommendation for Wilson disease. Supported with an ESCA (in development). |
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Sebelipase alfa (Kanuma®) |
For Wolman disease |
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Sodium benzoate injection | ||
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Sodium benzoate SF liquid | ||
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Sodium benzoate tablets | ||
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Sodium phenylbutyrate injection | ||
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Sodium phenylbutyrate tablets | ||
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Sucroferric oxyhydride |
Under review. |
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Trientine Dihydrochloride (Cufence®) |
NHS Commissioned |
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Trientine Tetrahydrochloride (Cuprior®) |
NHSE commissioned |
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Trometamol 7% injection (50ml) | ||
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Ubidecarenone | ||
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Velaglucerase alfa injection |
NHSE commissioned. |
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Velmanase alfa (Lamzede®) |
For alpha-mannosidosis |
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Zinc Acetate (WILZIN®) |
