National Spasmodic Dysphonia Association

Dear Dystonia Researchers and Friends,

As you are most likely aware the Office of Rare Diseases and several Institutes at the NIH have recently released an announcement entitled: Rare Diseases Clinical Research Consortia (RDCRC) for the Rare Diseases Clinical Research Network (U54) . The announcement is a reissue of an RFA which was previously released in 2003 and that led to establishment of several very successful rare disease clinical consortia and networks. The goal of such networks is to facilitate clinical research in rare diseases. Support will be provided for collaborative clinical research, including longitudinal and clinical studies and/or phase I , II and II/III trials; training of clinical investigators; pilot and demonstration projects; a test bed for clinical data management; and access to information related to rare diseases. Related initiatives, some of which will be a part of the application include a biorepository (cell and DNA), a clinical/genetic registry and database, and an animal model testing center. More details about this announcement can be found by clicking here.
The letter of intent receipt date for the application is July 20, 2008, and the application receipt date is August 20, 2008.

Since the dystonias are rare diseases, the RFA provides an unusual opportunity of major importance to the dystonia community. One of the integral and required elements of the RFA is the participation of patient support organizations, both in the process of preparing and submitting the application, as well as in the course of the funded clinical research work. As stated in the Announcement: “It is important to indicate prior collaborative arrangements between investigators in the group and patient support organizations, to emphasize the events that have led to the current application.” Discussions with NIH program staff have made it clear that more than a single application would be a mistake and seen as a division in the research and advocacy community. Therefore, the signatories of this letter representing all major dystonia foundations and support groups have agreed to strongly support the submission of such an application on behalf of the entire dystonia community. Informal planning and exploratory discussions are now underway. It is our strong belief that, considering a very competitive nature of the application process, we have to maximize our potential for success by supporting the strongest possible application. Our intent is to lend full support to the initiative, in order to maximize the chances of success. While the competition will be considerable and success is not guaranteed, it is clear that this undertaking will open up further opportunities and greatly strengthen the dystonia field overall.

At this point we would like to know if you are interested in participating in this effort as a potential participant or as a supporter of this initiative in any form. After we hear from you, we will establish a small working group that will involve prospective principal investigators and project leaders as well as representatives of all the foundations. We intend to communicate through conference calls and face to face meetings, as needed. We will strive to provide as much support and advice as possible to those who will take the lead in preparing and submitting the application.

Clearly, what is at stake here is a unique opportunity to propel dystonia clinical research to another level in our continuous struggle to find better treatments and ultimately a cure. We must, as a community, take concerted action to secure precious resources for the establishment of a dystonia clinical research consortium and network. This opportunity will not be made available again for at least five years. Since time is of the essence we ask you to respond at your earliest convenience but no later than March 24, 2008. Please contact Kimberly Kuman at the NSDA ([email protected]) for questions and confirmation of your interest in this initiative.

Christy Ludlow, Ph.D.
Scientific Director, National Spasmodic Dysphonia Association

Susan Bressman, M.D.
President, WE MOVE

Cynthia Comella, M.D.
Chair, Dystonia Study Group

Ted Dawson, MD, Ph.D.
Scientific Director, Bachmann-Strauss Dystonia and Parkinson’s Foundation

Mahlon DeLong, M.D.
Scientific Director, Dystonia Medical Research Foundation

Mark Hallett, M.D.
Scientific Director, Benign Essential Blepharospasm Research Foundation
National Spasmodic Dysphonia Association

E-mail: [email protected]
Phone: 800-795-6732


March 19, 2008