David Warnock, MD, Professor of Medicine (Emeritus), University of Alabama at Birmingham, discusses recent advances in Fabry disease clinical research.
Fabry disease is a rare genetic, multisystemic lysosomal disease characterized by specific cutaneous, neurological, renal, cardiovascular, cochleo-vestibular, and cerebrovascular manifestations.
Dr. Warnock describes recent advancements in the treatment of Fabry disease, including two enzyme treatments and a chaperone therapy. While these treatment options provide hope for patients and a base for research, there are still unmet clinical needs in this population.
For more information on the GRIDS Symposium, click here.
To learn more about rare lysosomal storage diseases, visit https://checkrare.com/diseases/lysosomal-storage-disorders/