Group Aims to Understand and Find Effective Treatments for Rare Disorders

FD is a unique and very rare disorder, but it is also part of a larger group of autonomic disorders that has recently attracted significant interest and funding from the NIH. A national research consortium comprising experts from around the country is focusing on these disorders. The consortium operates out of several prestigious institutions, including NYU, Vanderbilt, Harvard, the Mayo Clinic and the NIH. Dr. Horacio Kaufmann of the NYU Dysautonomia Center is one of the six experts who are members of the consortium, and the Dysautonomia Foundation is a participating support group.
 
Wendy R. Galpern, MD, PhD (Program Director, Clinical Trials, NINDS / NIH) explains the origins and aims of the program:
 
"In order to directly address the research needs in rare disorders, the Office of Rare Disease Research (ORDR) of the National Institutes of Health (NIH) initiated the Rare Diseases Clinical Research Network (RDCRN) in 2003 in collaboration with the NIH Institutes and Centers, including the National Institute of Neurological Disorders and Stroke (NINDS). The objectives of the RDCRN are to provide a specialized infrastructure to support research in the rare diseases and to promote collaboration across centers in order to advance our understanding of rare diseases and advance therapeutic development.

Each consortium collaborates with patient advocacy groups at the consortium and network level and has a major component related to the training of new clinical research investigators.
 
The RDCRN was expanded in October 2009 and now includes 19 research consortia, four of which have studies... aimed at exploring many components of these disorders including natural history, epidemiology, genetics, diagnosis, and treatment."
 
The Autonomic Disorders Consortium (ADC) was established as a Rare Disorders Clinical Research Consortium (RDCRC) in August 2009. The team includes physicians, scientists, nurses, patients and support groups dedicated to finding new therapies to treat and cure these diseases. 
 
The Autonomic Disorders Consortium, centered at Vanderbilt University and run by Dr. David Robertson, joins with patient support groups to harness the knowledge and energies of physicians and investigators in the major centers where these patients are cared for, so that they can discover ways to treat and to cure these diseases. The greater the collaboration between doctors and patients, the more we can learn about these disorders. This important first step is necessary if we are ever to find genuinely effective and curative treatments. All consortium sites (Mayo, Harvard, New York University, The National Institutes of Health and Vanderbilt) have long traditions in discovery and treatment of autonomic disorders.