For the rare diseases clinical research networkMolecular Genetics and MetabolismGriggs RC, Batshaw M, Dunkle M, Gopal-Srivastava R, Kaye E, Krischer J, et al. Clinical research for rare disease: Opportunities,
The Rare Diseases Clinical Research Network (RDCRN) Contact Registry has grown in size and scope since it was first reported in this journal in 2007. In this paper, we reflect on our seven years' experience developing and expanding the RDCRN Contact Registry to include many more rare diseases....
two catalogs of rare diseases cover 207 conditions, but more than 90% of these still lack effective treatment options. The Shenzhen Rare Disease Clinical Medical Research Center is poised to tackle these challenges head-on by advancing research and diagnosis, and by seeking...
two catalogs of rare diseases cover 207 conditions, but more than 90% of these still lack effective treatment options. The Shenzhen Rare Disease Clinical Medical Research Center is poised to tackle these challenges head-on by a...
Clinical delivery expertise:Our team includes highly experienced Clinical Research Associates (CRAs) and Patient Leads (PLs) with specialized expertise in rare diseases across all major regions. Passion and commitment:Our dedicated team is committed to improving the lives of rare disease patients, ensuri...
The National Institutes of Health (NIH) established the Rare Diseases Clinical Research Network to address the unique challenges of performing research on rare diseases. The Urea Cycle Disorders Consortium (UCDC) was one of the original ten consortia established. The UCDC represents a unique partnershi...
National Human Genome Research Institute. Rare genetic diseases. Available here. Accessed December 2024. EveryLife Foundation for Rare Diseases. Challenges to diversity in rare diseases. Available here. Accessed December 2024. Rare Disease Diversity Coalition. Inequities in the rare disease community. Ava...
Because these diseases affect such a small group of people, it can be difficult to gather enough people for a clinical trial even with the Rare Disease Clinical Research Network is in place. Kelly explained that while heart disease studies may have tens of thousands of patients, the best a ...
Decentralized clinical trials Data licensing Patient recruitment Commercial alerts Pack Health® Mobile phlebotomy Expertise Rare diseases Cell & gene therapy Oncology Neurology Cardiometabolic More therapeutic areas Life Sciences Pharma Biotech Contract research organizations (CROs) ...
Among the unique features of the Rare Diseases Clinical Research Network (RDCRN) Program is the requirement for each Consortium to include patient advocacy groups (PAGs) as research partners. This development has transformed the work of the RDCRN and is a model for collaborative research. This art...