The North American Pediatric Renal Trials and Collaborative Studies and Horizon Pharma Announce New Long-Term Cystinosis Registry

A prospective longitudinal natural history registry designed to provide long-term data evaluating people living with cystinosis was launched today at a meeting of The North American Pediatric Renal Trials and Collaborative Studies (NAPRTCS) in Chicago, Ill. The registry will collect and organize physician-reported data that will help healthcare professionals better understand the natural history of cystinosis. Data collected will also be available to researchers exploring new potential treatments for cystinosis. Utilizing the established NAPRTCS infrastructure which collects data on patients with chronic kidney disease, on dialysis and following kidney transplant, the cystinosis registry was made possible through a partnership between NAPRTCS, leading physicians in the cystinosis community, and the medical leadership at Horizon Pharma plc (Nasdaq: HZNP).

“The registry will also help us answer a number of questions related to the long-term outcomes for patients and the long-term effects of cystinosis on the kidneys and all other organ systems.”

“Initial cystinosis natural history studies were conducted decades ago by the National Institutes of Health and there have been few studies since that time that track the progression of cystinosis,” said Bradley Warady, M.D., professor of pediatrics at the University of Missouri-Kansas City School of Medicine, director of pediatric nephrology at Children’s Mercy Kansas City and member of the NAPRTCS board of directors. “With recent advancements, people with cystinosis are living longer lives; however, our understanding of the disease in the context of these recent advancements and new treatments is limited. By gathering data from people living with cystinosis over an extended period of time, the NAPRTCS registry will provide key information for physicians and researchers.”

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