Newsletter of the NIH Rare Diseases Clinical Research Network
Volume 3, Issue 1. January 2012

www.RareDiseasesNetwork.org

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In This Issue

Message from Alan Percy, MD – Newly re-elected Chair of the Rare Diseases Clinical Research Network

Spotlight on RDCRN Consortia: The Vasculitis Clinical Research Consortium

Arginine Therapy in Argininosuccinic Aciduria and its Effects on Liver Dysfunction –Evidence Based Analysis from a Randomized Clinical Trial

Lessons from the MILES Trial

National Center for Advancing Translational Sciences (NCATS) Established

Collaboration Between RDCRN & CTSA at The University of Kansas Medical Center

The Ride for Rare Diseases: A Model for Consortia Cooperation

STAIR PI Steiner Named Deputy Editor for Genetics in Medicine

RDCRN Opens Over 20 New Studies for Patient Accrual

About Spotlight

Past Issues

Editorial Board

 

Rare Diseases Headlines:

Office of Rare Diseases Research launches web site for Rare Diseases Human Biorepostiories and Biospecimens (RD-HUB)
>> More

Stewardship and Public Access to Research Data
>> More

RDCRN Conditions Approved for Social Security Compassionate Allowance
>> More

Featured Event:

Rare Diseases Day 2012
>> More

Spotlight Announcements / Calendar of Events

Lysosomal Disease Network: WORLD Symposium
San Diego, CA
Feb 8-10, 2012

Feedback

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.

Looking Forward

Message from Alan Percy, MD
Newly re-elected Chair of the Rare Diseases Clinical Research Network


Alan K. Percy, M.D.
Chair, RDCRN Steering Committee

As we enter the coming year, I wish everyone in the Rare Disease Clinical Research Network my very best wishes and thank you for your continued support of my role as Steering Committee chair. It is a great pleasure to see this group of investigators across the globe and centered at the NIH making such wonderful progress over the now eight years of experiences for many.

We certainly need to remember all the participants in this enterprise, particularly the many individuals and families whom we serve, the patient advocacy groups who provide such a vital role, and the many interested groups who support us in these endeavors.

While we work diligently in our individual silos, we must be mindful that the DMCC is engaged with every study, a remarkable effort under the expert guidance of Jeff Krischer. Without doubt, we would not be where we are today if not for the leadership and longstanding support of Steve Groft and the members of his ORDR team. I am certain of this.

We have much work to do as a network. I look forward to the next year and once again will be asking each investigative team to be certain that they participate actively in the standing committee structure as currently set forth. In this manner, I am certain that we will be able to maintain and advance the active support of NIH specifically and the public more generally in the rare disorders that we represent.