Quarterly Newsletter of the NIH Rare Diseases Clinical Research Network
December 2010

www.RareDiseasesNetwork.org

In This Issue

Introducing Spotlight on Rare Diseases

The NIH Rare Diseases Clinical Research Network (RDCRN) Doubles in Size

Message from Alan Percy, RDCRN Steering Committee Chair

About Spotlight

Current Issue of Spotlight

Network News and Events

World Rare Disease Day
February 28, 2011
rarediseaseday.us

7th Annual World Symposium, Research for Lysosomal Diseases
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) >> 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

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Send comments to RDNWebmaster@epi.usf.edu

Message from Alan Percy, 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.

Second, at the outset, we committed to establishing committees to address issues and opportunities confronting each consortium based on the experiences of the so-called legacy consortia from the first funding cycle. These committees are the Strategic Planning Committee (Peter Merkel, Chair), the Registry Committee (Rachel Richesson, Chair), the Training Committee (Robert Steiner, Chair), and the Operations Committee (Michael Knowles, Chair).

Third, the September in-person Steering Committee meeting was preceded by the second Conference on Clinical Research for Rare Diseases. This was a terrific opportunity for trainees to present their work and to learn from the impressive faculty and for Network investigators to understand the activities of each consortium. A full update on the conference appears elsewhere in this issue of Spotlight.

 

 

Alan Percy, MD
Professor, University of Alabama at Birmingham
PI of Angelman, Rett and Prader-Willi Syndromes Consortium
Chair, RDCRN Steering Committee