Agalsidase beta therapy for advanced fabry disease: A randomized trial

Fabry disease is a rare, X-linked lysosomal storage disorder that can cause early death from renal, cardiac, and cerebrovascular involvement. In this double-blind multicenter trial, 82 adults with kidney dysfunction from Fabry disease were randomly assigned to bimonthly infusions of enzyme replacement with agalsidase beta or placebo for up to 35 months. Agalsidase beta reduced the frequency of and time to renal, cardiac, and cerebrovascular events and death and caused infusion reactions more often than placeb