Spotlight - Volume 4, Issue 1. Spring 2013
In this issue of Spotlight…
The purpose of Spotlight on Rare Diseases is to highlight the activities and progress of the Rare Diseases Clinical Research Network (RDCRN) and the Patient Advocacy Groups (PAGs) that collaborate with RDCRN investigators in elucidating the mechanisms of rare diseases and discover of therapies for them. This e unique program is sponsored by the NIH Office of Rare Diseases Research (ORDR) at the National Center for Advancing Translational Science (NCATS in collaboration with eight Institutes (NINDS,NIAID, NIAMS, NIDDK, NIDCR, NICHD, NHLBI and NCI.
The RDCRN currently comprises 17 unique Consortia and a Data Management and Coordinating Center (DMCC) involving groups of investigators collaborating with each other, and also with PAGs in close and often intense collaboration. The close relation between rare disease investigators and PAGs is a unique strategy by which progress and mutual interaction ensure optimal planning and execution of projects. Each rare disease consortium represents one or more groups of diseases or special disciplines, all supported by the efficient and overarching structure of the RDCRN DMCC at the University of South Florida.
In this issue, there are contributions from the Rare Kidney Stone Consortium (RKSC) and the Brain Vascular Malformation Consortium (BVMC), the latter describing a recent meeting organized by the BVMC and the PAGs supporting it, in an article contributed by Amy Akers, Ph.D., Chief Scientific Officer of the Angioma Alliance.
This issue also provides updates on the currently open research projects and clinical trials throughout the 17 RDCRN consortia, providing evidence of the great productivity of this program in disease pathophysiology, novel therapeutic entities, and a robust repurposing of drugs which characterizes this program.
RDCRN Research Studies
For a list of the protocols of the RDCRN, please visit the website below for a full listing of all RDCRN studies: www.rdcrn.org/studies
Spotlight is edited by David Robertson, MD, Elton Yates Professor of Medicine, Pharmacology and Neurology and Director of the Clinical and Translational Research Center of Vanderbilt University's Clinical and Translational Science Award (CTSA). Dr. Robertson is PI of the Autonomic Disorders Consortium. Cindy Dorminy, MEd LPN is Assistant Editor.
The staff of Spotlight are enthusiastic about the potential of our new publication to enhance communication among those interested in treatment and cure of rare diseases. We are very interested in your thoughts about how Spotlight can be improved. If you have suggestions for future issues, please let us hear from you. You can send your comments to RDNWebmaster@epi.usf.edu.