Subject: FW: Spotlight on Rare Diseases

Quarterly Newsletter of the NIH Rare Diseases Clinical Research Network

December 2010

Introducing Spotlight on Rare Diseases Welcome to the inaugural issue of Spotlight on Rare Diseases, the eNewsletter that highlights the mission and dynamism of the NIH Rare Diseases Clinical Research Network (RDCRN). The purpose of Spotlight on Rare Diseases is to provide a single location for information on the activities and achievements of the RDCRN. Personnel within the NIH and other funding institutions will be able to review the valuable work in rare diseases research of all RDCRN investigators in one publication. Spotlight will also serve to educate members of the rare disease community who are not part of the Network, so that they better understand the RDCRN model and thus allow investigators whose patient constituencies are not yet represented in the RDCRN to be in a better position to compete successfully to become a part of the Network. >> More The NIH Rare Diseases Clinical Research Network (RDCRN) Doubles in Size The NIH Rare Diseases Clinical Research Network (RDCRN) comprises 19 consortia involving some 80 investigators at over 70 institutions. This program and its Data Management Coordinating Center (DMCC) are receiving an aggregate total of $117 million over five years. The research conducted with the new funding will explore the natural history, epidemiology, diagnosis, and treatment of more than 95 rare diseases. "The progress made by researchers through the Network over the past six years is important and impressive," said NIH Director Francis S. Collins, M.D., Ph.D. in announcing the expansion of the RDCRN "We have shown that this approach can be a catalyst for progress in meeting the challenge of rare diseases." >> More Message from Alan Percy, MD, RDCRN Steering Committee Chair As we enter year two of the current Rare Diseases Clinical Research Network, a number of important accomplishments should be emphasized. First, the inaugural issue of Spotlight on Rare Diseases under David Robertson's editorship is an opportunity to highlight our individual and collective progress, underscore the importance of rare diseases research, and emphasize the collaborative efforts of this Network of widely diverse disorders. This is our chance to show our federal sponsors, patient advocacy groups, and the American public that orphan diseases are being taken seriously and that our ultimate goal is engagement in clinical trials to provide safe and effective interventions. While we work in our respective silos, the Network is a critical key to promoting the notion that the sum of our efforts exceeds that of the individual consortia. >> More

Network News and Events World Rare Disease Day February 28, 2011 rarediseaseday.us 7th Annual World Symposium, Research for Lysosomal Diseases Las Vegas, Nevada February 16-18, 2011 5th International Dystonia Symposium Barcelona, Spain October 20-22 2011 Featured Event: 2nd Annual Conference on Clinical Research for Rare Diseases (CCRRD) The Rare Diseases Clinical Research Network (RDCRN) and the Clinical and Translational Science Awards (CTSA) program co-sponsored the 2nd Conference on Clinical Research for Rare Diseases (CCRRD) which was held on Tuesday September 21, 2010 in Bethesda, Maryland. The Conference was again planned and organized by Dr. Peter Merkel of the Boston University School of Medicine. This unique conference focused on research methodology for rare diseases and with a particular emphasis on information helpful for trainees and junior faculty engaged in such research. There were 270 meeting attendees, including trainees, junior and senior faculty, NIH program officials, and leaders of patient advocacy groups. >> More Rare Diseases Headlines: President Signs "Improving Access to Clinical Trials Act" into law >> More CTSA Consortium to Enhance Support of Rare Diseases Research >> More IOM Releases Report on Accelerating Rare Diseases Research >> More

www.RareDiseasesNetwork.org	The Rare Diseases Clinical Research Network (RDCRN) was established by the National Institutes of Health (NIH) to develop research studies for rare diseases, and to encourage cooperative partnerships among researchers at over 150 clinical centers around the world. This increased cooperation may lead to discoveries that will help treat and perhaps prevent these rare diseases, as well as produce medical advances that will benefit the population in general. The Rare Diseases Clinical Research Network is comprised of a Data Management Coordinating Center and 19 consortia studying over 100 rare diseases.
		Funding and/or programmatic support for the Rare Diseases Clinical Research Network (RDCRN) has been provided by NIH Office of Rare Diseases Research (ORDR).
The National Institutes of Health does not endorse or recommend any commercial products, processes, or services. The views expressed in written materials or publications do not necessarily reflect the official policies of the Department of Health and Human Services; nor does mention by trade names, commercial practices, or organizations imply endorsement by the U.S. Government.