The Future of Treatment for Patients With Fabry Disease (FD) and Renal Complications

Target Audience
This educational activity is directed toward nephrologists, cardiologists, gastroenterologists, geneticists, genetic counselors, and any other health care professionals involved in the diagnosis or management of Fabry disease.

Activity Overview
Due to the broad spectrum of phenotypes of Fabry disease and similarities with other disorders, diagnosis is challenging and often delayed. The primary cause of late morbidity and premature mortality in patients with Fabry disease is the irreversible damage that can occur without treatment; therefore, early, definitive diagnosis and timely treatment are crucial. Our overall understanding of Fabry disease has advanced, but greater knowledge of pathophysiological mechanisms, disease manifestations, and effective treatment is needed to improve outcomes among the various Fabry disease subgroups.

This program will look at the differential diagnosis, testing algorithms, and genetics underlying Fabry disease. Expert faculty will review the efficacy and safety of current therapies, with a focus on renal complications, and the results of studies of emerging therapies aimed at improving symptoms, preventing disease progression, and improving patient quality of life.

Learning Objectives
Upon completion of this activity, you should be better able to:

Describe the pathophysiology of Fabry disease with respect to the development of renal complications
Develop a differential diagnosis for patients presenting with symptoms consistent with Fabry disease
Evaluate the efficacy, safety, and limitations of current and emerging therapies for patients with Fabry disease and renal complications
Apply clinical trial data and best practice guidelines to the long-term, multidisciplinary management of Fabry disease