A place where you can access information about how to help manage your patients
Achondroplasia
Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2)
Hemophilia A
Phenylketonuria (PKU)
BRINEURA®(cerliponase alfa)
NAGLAZYME®(galsulfase)
PALYNZIQ®(pegvaliase-pqpz) Injection
ROCTAVIANTM(valoctocogene roxaparvovec-rvox)
VIMIZIM®(elosulfase alfa)
VOXZOGO®(vosoritide)
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