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

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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.

National Center for Advancing Translational Sciences (NCATS) Established

Major Win for NIH Director Francis Collins and for Rare Diseases Research


Francis S. Collins, M.D., Ph.D
Director, National Institutes of Health

Days before the 2012 New Year, Congress passed and the President signed landmark legislation championed by NIH Director Francis Collins, aimed at transforming the way human research is done. Key leaders in NIH, investigators at the nations academic medical centers,  the Food and Drug Administration, and even much of the pharmaceutical sector saw NCATS as a bold new structure that would change the way research is done.

A total of $576.5 million was appropriated this year to fund the new National Center for Advancing Translational Sciences (NCATS), and of that the largest program at NCATS will be the Clinical and Translational Science Awards (CTSAs), which receive $487.8 million of the new institute's budget. The CTSAs provide the infrastructures to conduct research in human subjects in an optimal and efficient environment through a national consortium of 60 medical research institutions working together to improve the way clinical and translational research is conducted nationwide .

 “Congressional support for the National Center for Advancing Translational Sciences (NCATS) marks a major milestone in mobilizing the community effort required to revolutionize the science of translation,” said NIH Director Collins in NIH News on December 23, 2011.

“Patients suffering from debilitating and life threatening diseases do not have the luxury to wait the 13 years it currently takes to translate new scientific discoveries into treatments that could save or improve the quality of their lives. The entire community must work together to forge a new paradigm, and NCATS aims to catalyze this effort.”

NCATS will serve as the home of many programs that were previously dispersed across the NIH in other venues. These include (1) the Office of Rare Diseases Research, (2) the CTSAs, (3) the Therapeutics for Rare and Neglected Diseases (TRND) program, and (4) the Cures Acceleration Network (CAN), which enables NCATS to fund research in new and innovative ways.

The Office of Rare Diseases Research (ORDR) and the CTSAs particularly expect to work together fruitfully to fulfill the mandate to increase focus on rare diseases.

Other components that comprise NCATS include: