Braun, Fabian; Abed, Ahmed; Sellung, Dominik; Rogg, Manuel; Woidy, Mathias; Eikrem, Øystein Solberg; Wanner, Nicola; Gambardella, Jessica; Laufer, Sandra; Haas, Fabian; Wong, Milagros; Dumoulin, Bernhard; Rischke, Paula; Mühlig, Anne; Sachs, Wiebke; von Cossel, Katharina M; Schulz, Kristina; Muschol, Nicole; Gersting, Sören; Muntau, Ania C.; Kretz, Oliver; Hahn, Oliver; Rinschen, Markus; Mauer, Michael; Bork, Tillmann; Grahammer, Florian; Liang, Wei; Eierhoff, Thorsten; Römer, Winfried; Hansen, Arne; Meyer-Schwesinger, Catherine; Iaccarino, Guido; Tøndel, Camilla; Marti, Hans Peter; Najafian, Behzad; Puelles, Victor G; Schell, Christoph; Huber, Tobias B (Journal article; Peer reviewed, 2023)
Current therapies for Fabry disease are based on reversing intracellular accumulation of globotriaosylceramide (Gb3) by enzyme replacement therapy (ERT) or chaperone-mediated stabilization of the defective enzyme, thereby ...