Blar i Department of Clinical Medicine på forfatter "van der Veen, Sanne"
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Early start of enzyme replacement therapy in pediatric male patients with classical Fabry disease is associated with attenuated disease progression
van der Veen, Sanne; Körver, Simon; Hirsch, A; Hollak, Carla E.M.; Wijburg, Frits A; Brands, M; Tøndel, Camilla; Van Kuilenburg, André B.P.; Langeveld, Miriam (Journal article; Peer reviewed, 2022)Background Enzyme replacement therapy (ERT) slows disease progression of Fabry disease (FD), especially when initiated before the onset of irreversible organ damage. However, with the clinically asymptomatic progression ...