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ZOLGENSMA ( Onasemnogene abeparvovec )
ZAVESCA ( Miglustat )
VIMIZIM VIMZIM ( Elosulfase alfa )
SKYCLARYS (omaveloxolone)
ORKAMBI ( lumacaftor- ivacaftor )
NUPLAZID ( Pimavanserin )
ELAPRASE ( Idursulfase )
NGENLA (somatrogon-ghla) injection
CARBAGLU ( carglumic acid )
CERDELGA ( ELIGLUSTAT )
INGREZZA ( Valbenazine )
XERMELO ( telotristat ethyl )
Ultomiris (ravulizumab)
Lupkynis ( Voclosporin )
Xphozah ( Tenapanor)