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- Title
Long-term outcome of enzyme-replacement therapy in advanced Fabry disease: evidence for disease progression towards serious complications.
- Authors
Weidemann, F; Niemann, M; Störk, S; Breunig, F; Beer, M; Sommer, C; Herrmann, S; Ertl, G; Wanner, C
- Abstract
The long-term effects of enzyme-replacement therapy (ERT) in Fabry disease are unknown. Thus, the aim of this study was to determine whether ERT in patients with advanced Fabry disease affects progression towards 'hard' clinical end-points in comparison with the natural course of the disease.
- Publication
Journal of internal medicine, 2013, Vol 274, Issue 4, p331
- ISSN
1365-2796
- Publication type
Journal Article
- DOI
10.1111/joim.12077