Sanford Research Vice President David Pearce, PhD, with the Sanford Children’s International Board
At Sanford Research, scientists and staff have developed a centralized registry to work with physicians, scientists and organizations around the world to collect data on these rare diseases. This will provide important information in linking these diseases and supporting novel therapies and cures. The mission of the Sanford Children’s Health Research Center is to develop new therapies from discoveries made in research labs by integrating basic science with clinical practice, a so-called “bench-to-bedside” approach. Dr. David Pearce, director of the Children’s Health Research Center and vice president of Sanford Research, has a track record of using interdisciplinary research to study the neurodegenerative disorder, Batten disease. This same interdisciplinary approach to research currently applies to a number of childhood diseases, many of which are categorized as rare. Many new developments in the diagnosis and treatment of human disease rely on research that involves
the collection and analysis of data. Lack of resources to apply to clinical studies, and the spread of rare patients around the world, limits the amount of data available for these rare diseases. The CoRDS registry collects and collates contact and clinical information. Even the most basic information – What is the diagnosis? When and where was the diagnosis made? Is the individual interested in participating in research? – can be extremely useful to researchers. This data collection, and central repository for patient and disease information, is a core focal point of the Sanford Children’s Health Research Center. Through the engagement of patient advocacy organizations, and communication to those organizations’ members, CoRDS provides a way for patients to learn about research opportunities. This allows for greater clinical trial participation which then has a greater potential impact through discovery.
are often motivated to participate in research, but there are so few opportunities,” said Pearce. “The CoRDS registry will support the acceleration of this research by providing a resource through which researchers can access information and identify individuals who are interested in participating.” Nearly 60 rare disease organizations have partnered with Sanford and the list is growing. The CoRDS registry is open to individuals of any age who have been diagnosed with any rare disease and includes participants from around the world. “Collaboration is paramount in this field,” says Liz Donohue, director of CoRDS. “This means engaging patient advocacy organizations, rare disease researchers, and healthcare providers.” For more information, visit sanfordresearch.org/cords/.
“Patients affected by rare diseases
Of the 7,000 known rare diseases, only 400 have FDA approved therapies. 80% of these are diseases that are genetic, and 50% of those affected are children. Many of these children
do not survive beyond the age of 4. 4 | discoveries