A2 Refereed review article in a scientific journal

Screening, diagnosis, and management of patients with Fabry disease: conlusions from a "Kidney Disease: Improving Global Outcomes" (KDIGO) controversies conference




AuthorsSchiffmann R, Hughess DA, Linthorst GE, Ortiz A, Svarstad E, Warnock DG, West ML, Wanner C; for Conference Participants

Publication year2017

JournalKidney International

Volume91

Issue2

Number of pages10

ISSN0085-2538

DOIhttps://doi.org/10.1016/j.kint.2016.10.004


Abstract

Patients with Fabry disease (FD) are at a high risk for developing chronic kidney disease and cardiovascular disease. The availability of specific but costly therapy has elevated the profile of this rare condition. This KDIGO conference addressed controversial areas in the diagnosis, screening, and management of FD, and included enzyme replacement therapy and nonspecific standard-of-care therapy for the various manifestations of FD. Despite marked advances in patient care and improved overall outlook, there is a need to better understand the pathogenesis of this glycosphingolipidosis and to determine the appropriate age to initiate therapy in all types of patients. The need to develop more effective specific therapies was also emphasized.



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