Website Edition: June/July 2011

DMRF Provides Essential Services for Physicians and Patients

Resources Designed to Support Patient Care

By Mahlon R. DeLong, MD, Professor of Neurology, Emory School of Medicine
Scientific Director, Dystonia Medical Research Foundation

Lay patient advocacy organizations such as the Dystonia Medical Research Foundation (DMRF) fill a niche that no other entity can. Over the past 35 years, the DMRF has been able to engage basic and clinical scientists, increase awareness and understanding and, most importantly, stimulate and support research on a wide range of dystonia relevant topics. The DMRF serves a population of patients who historically suffered from frequent misdiagnosis, misunderstanding and a scarcity of treatments, and who now have a community of investigators, clinicians, institutions, and partners working together to make their lives better. To this end, the DMRF is an important resource for physicians because it offers a range of services and support programs for patients. By informing dystonia patients about the DMRF, physicians can help them to access the wide variety of programs available and better understand the scope of ongoing research efforts to find new treatments for dystonia.

The DMRF remains a major non-profit supporter of research on dystonia. A recent major accomplishment is its role in establishing joint programs with the National Institute of Neurological Disorders & Stroke (NINDS) in order to increase the scope of research on dystonia. The DMRF also serves as an administrative center for the Dystonia Coalition, a groundbreaking research collaboration with the Office of Rare Disorders, NINDS and several dystonia patient organizations as well as over 40 clinical centers dedicated to advancing translational research Recognizing the need for leadership in accelerating the development of more effective treatments, the DMRF has recently established a drug discovery and development program, the Cure Dystonia Initiative (CDI), whose goal is the development of new therapeutics and partnerships between academia, federal agencies and biopharmaceutical companies In addition the foundation has played a major role in sponsoring scientific meetings and workshops, recruiting and training Fellows and young investigators, providing patient and family support, and advocating in behalf of dystonia at the federal and NIH level.

The DMRF serves as an administrative center for the Dystonia Advocacy Network (DAN), the patient community's legislation and public policy watchdog in Washington, DC, which is actively engaged in matters that impact investigators, clinicians, and patients. The DAN advocates for increased federal funding. The foundation was successful in lobbying the Department of Defense to include dystonia as a condition eligible for funding in its Congressionally-directed, peer-reviewed medical research program, opening up a previously untapped funding opportunity for investigators. The DAN has also led the charge to create a regulatory path for follow-on biologics. As an example, when a coding issue threatened to complicate physician use and patient access to the recently approved therapeutic botulinum neurotoxin, Xeomin®, the DAN rallied representatives to meet with the Centers for Medicare and Medicaid Services to explain the ramifications. A unique temporary billing code was issued, resolving the matter. The DAN has also been a persistent voice for adequate physician reimbursement.

The onset of dystonia is often a life-changing event for patients. Coping with increasing disability and pain can be socially isolating and emotionally difficult. The DMRF offers a range of resources for patients with all forms of Dystonia. Information is available at the website given below. When dystonia patients contact the DMRF, they gain access to a reliable source of accurate information on dystonia, support groups, and connection to others through online social forums, patient meetings and webinars, as well as practical guidance for living as well as possible with dystonia. All of this contributes to improved overall disease management.

Since its establishment 35 years ago, the DMRF has become one of the finest organizations of its kind, serving the needs of patients and caregivers. In turn, affected individuals and their family members lead grassroots DMRF volunteer activities and local support groups. These volunteers are vital to the DMRF's success and function as important agents of public awareness, patient support, and fundraising in support of research-all this despite living with the daily struggles imposed by the disease. DMRF members are hungry for research updates, deeply appreciative of the work done by scientists contributing to the field, and frequently express how the broad scope of the DMRF's science program give them hope, which fuels their resilience.

Readers can learn more about the DMRF at www.dystonia-foundation.org. Click "Dystonia Research" to learn more about the science program and funding opportunities. For a special look at how dystonia impacts the lives of patients in their own words, visit www.dystonia-foundation.org/faces_of_dystonia.

Mahlon R. DeLongAbout Mahlon R. DeLong, MD
Mahlon R. DeLong, MD is a Professor of Neurology at Emory University School of Medicine. Among numerous awards, Dr. DeLong received a Javitz Neuroscience Investigator Award from the NINDS for his research, the Schneider Lecture by the American Association of Neurological Surgeons, and the American Academy of Neurology Wartenburg Award. Dr. DeLong was selected as the first recipient of the Deans Distinguished Faculty Award and Lecturer at Emory and is an elected member of the Johns Hopkins Society of Scholars, the Institute of Medicine and the American Academy of Arts and Sciences. He served on the Advisory Council of the NINDS from 1996-2000 and as a Fellow of the AAAS of which he currently serves as Chair of the Section of Neuroscience. He has served as the Scientific Director of the Dystonia Medical Research Foundation since 1994.

Dr. DeLong has played a major role in clarifying the functional organization of the basal ganglia and the role of these structures in movement and the pathophysiology of movement disorders. Dr. DeLong's research contributed significantly to the revival of and development of new and more effective surgical targets and approaches for the treatment of Parkinson's Disease and other movement disorders.