Clinical trials are studies to develop, produce and evaluate the effectiveness of new treatments and therapies for diseases.
Craig B. Langman, MD, has a long-standing interest in genetic bone and kidney stone disease. His current work focuses on the translational aspects of genetic bases of these diseases and the pharmacogenetics of specific bone dysplasias.
Jerome C. Lane, MD, has major areas of clinical interest including pathogenesis of hemolytic-uremic syndrome (HUS), and mechanisms of clinical progression in congenital renal disease and urogenital malformations. In addition, Dr. Lane has a considerable background and laboratory experience in renal transport of major ions.
This is a long-term, open-label study of the safety, tolerability and effectiveness of RP103 in cystinosis patients who are naïve to any form of cysteamine treatment. Subjects will receive RP103 treat...