Reduction of podocyte globotriaosylceramide content in adult male patients with Fabry disease with amenable GLA mutations following 6 months of migalastat treatment

Conclusion
Migalastat treatment of 6 months duration in eight male patients with Fabry disease demonstrated effective GL3 clearance from the podocyte, an important and relatively ERT-resistant glomerular cell.

Source: Journal of Medical Genetics - October 23, 2017 Category: Genetics & Stem Cells Authors: Mauer, M., Sokolovskiy, A., Barth, J. A., Castelli, J. P., Williams, H. N., Benjamin, E. R., Najafian, B. Tags: Open access Therapeutics Source Type: research