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Inborn Errors of Metabolic Diseases (IEM)
Inborn Errors of Metabolic Diseases (IEM)
Aldurazyme (laronidase, Medical), Brineura (cerliponase alfa, Medical), Carbaglu (carglumic acid, Rx), Cerdelga (eliglustat, Rx), Cerezyme (imiglucerase Medical), Cholbam (cholic acid, Rx), Crysvita (burosumab-twza, Medical), Elaprase (idursulfase, Medical), Elelyso (taliglucerase alfa Medical), Fabrazyme (agalisidase beta, Medical), Galafold (migalastat, Rx), Kanuma (sebelipase alfa, Medical), Kuvan (sapropterin, Rx), Lumizyme (alglucosidase alfa, Medical), Mepsevii (vestronidase alfa-vjbk, Medical), Naglazyme (galsulfase, Medical), Nityr (nitisinone Rx), Orfadin (nitisinone, Rx), Palynziq,Ravicti (glycerol phenylbutyrate, Rx), Strensiq (asfotase alfa, Rx), Sucraid (sacrosidase, Rx), Vimizim (elosulfase alfa Medical), VPRIV (velaglucerase alfa Medical), Xuriden (uridine triacetate, Rx), Zavesca (miglustat Rx)

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