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Inborn Errors of Metabolic Diseases (IEM) & Rare Genetic Diseases
Inborn Errors of Metabolic Diseases (IEM) & Rare Genetic Diseases
Adagen (pegademase bovine, Rx), 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), Gamifant (emapalumab-lzsg, Medical), Kanuma (sebelipase alfa, Medical), Kuvan (sapropterin, Rx), Lumizyme (alglucosidase alfa, Medical), Mepsevii (vestronidase alfa-vjbk, Medical), Naglazyme (galsulfase, Medical), Nityr (nitisinone, Rx), Onpattro (patisiran, Medical), Orfadin (nitisinone, Rx), Palynziq (pegvaliase-pqpz, Rx), Ravicti (glycerol phenylbutyrate, Rx), Revcovi (elapegademase-lvlr, Rx), Strensiq (asfotase alfa, Rx), Sucraid (sacrosidase, Rx), Tegsedi (inotersen, Rx), Vimizim (elosulfase alfa Medical), VPRIV (velaglucerase alfa Medical), Xuriden (uridine triacetate, Rx), Zavesca (miglustat Rx)

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