DMRF Partners with Dystonia Coalition to Prepare for Clinical Trials
A recent article in the medical journal Neurotherapeutics provides a framework for testing new dystonia therapies in clinical trials. As researchers gain a better understanding of dystonia, new treatments and therapeutic approaches are being developed and must be tested in studies with human volunteers to prove they are safe and beneficial. Because dystonia is a complex disorder, the process for testing new therapies is equally complex. This paper addresses the opportunities and challenges inherent in clinical trials for dystonia, and helps prepare the field for implementing meaningful, well-designed studies.
The article stems from the Workshop on Designing Clinical Trials for Dystonia co-sponsored by the Dystonia Medical Research Foundation (DMRF) and the Dystonia Coalition, May 30-31, 2012 in Bethesda, Maryland. This workshop was unique because it brought together top dystonia clinicians, clinical trials experts, and biostatisticians to discuss the best approaches for designing clinical trials specifically for dystonia. The Director of the National Institutes of Health (NIH), Francis Collins, MD, PhD, referenced this meeting as an important advancement in a recent update to Members of Congress on dystonia research at NIH.
Funding for the original workshop was provided by a U13 grant from the National Institutes of Neurological Disorders and Stroke (NINDS) and the DMRF, with additional support provided by the Dystonia Coalition, which is funded through the national Office of Rare Diseases Research and NINDS.
The DMRF extends thanks and congratulations to all of the authors of the recent publication, with special appreciation to Dystonia Coalition Program Director, H. A. (Buz) Jinnah, MD, PhD; NIH/NINDS Division of Extramural Research Program Director, Wendy Galpern, MD, PhD; and DMRF Chief Scientific Officer Jan Teller, PhD.
Click the link below to view an abstract.
Designing Clinical Trials for Dystonia.
Galpern WR, Coffey CS, Albanese A, Cheung K, Comella CL, Ecklund DJ, Fahn S, Jankovic J, Kieburtz K, Lang AE, McDermott MP, Shefner JM, Teller JK, Thompson JL, Yeatts SD, Jinnah HA.
Neurotherapeutics. 2013 Nov 27. [Epub ahead of print]