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Inborn Errors of Metabolism & Rare Genetic Diseases (Self Administration)
Inborn Errors of Metabolism & Rare Genetic Diseases (Self Administration)
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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