Dystonia Medical Research Foundation

PROGRAM: SCIENCEISSUING 3 REQUESTS FOR APPLICATIONS FOR SCIENTIFIC RESEARCH; MANAGING THE PEER REVIEW PROCESS OF 30 SUBMITTED GRANT RESEARCH FELLOWSHIP AND CLINICAL FELLOWSHIP APPLICATIONS; SELECTING, FUNDING AND MANAGING 10 RESEARCH GRANTS, 3 RESEARCH FELLOWSHIPS AND 1 CLINICAL FELLOWSHIP, ALL AIMED AT CREATING A BETTER UNDERSTANDING OF THE PATHOPHYSIOLOGY OF DYSTONIA, IDENTIFYING AND MODIFYING CARRIER GENES, AND DEVELOPING IMPROVED TREATMENTS AND ULTIMATELY A CURE FOR DYSTONIA; ANDMEETING WITH PHARMACOLOGICAL AND BIOTECH COMPANIES TO ENGAGE THEM IN THE DEVELOPMENTOF TREATMENTS FOR DYSTONIA; ANDHOSTING, PLANNING AND SPONSORING THE SAMUEL BELZBERG 6TH INTERNATIONAL DYSTONIA SYMPOSIUM IN JUNE 2023; ANDCONTINUED FUNDING OF A CLINICAL FELLOWSHIP PROGRAM DESIGNED TO SUPPORT TRAINING OF PHYSICIANS IN PREPARATION FOR A CLINICAL AND/OR RESEARCH CAREER IN MOVEMENT DISORDERS WITH SPECIAL FOCUS ON DYSTONIA; ANDSPONSORING AND PARTICIPATING IN DOMESTIC AND INTERNATIONAL SCIENTIFIC MEETINGS INCLUDING THE INTERNATIONAL PARKINSON'S AND MOVEMENT DISORDER SOCIETY'S CONGRESS; DYSTONIA CONSENSUS MEETING; AMERICAN NEUROLOGISTS ANNUAL MEETING; THE INTERNATIONAL ASSOCIATION OF PARKINSONS; RELATED DISORDERS ANNUAL MEETING; AND NON-MOTOR SYMPTOMS OF DYSTONIA WORKSHOP.HOSTING A MEETING OF THE NON-MOTOR SYMPTOMS OF DYSTONIA WORKING GROUP TO SUPPORT EFFORTS IN PUBLISHING A PAPER ON THE TOPIC. ORGANIZING AND HOSTING A MEETING OF 14 EARLY STAGE INVESTIGATORS TO SUPPORT THEIR EFFORTS TO WORK COLLABORATIVELY AND BUILD SCIENTIFIC WORKSHOPS.REVIEWING CURRENT RESEARCH DATA AND EXPLORING STUDIES NEEDED TO ACCELERATE IMPROVED TREATMENTS; ANDSUPPORTING AND MAINTAINING THE AVAILABILITY OF RESOURCES TO ACCELERATE DYSTONIA RESEARCH GLOBALLY SUCH AS THE DYSTONIA BRAIN COLLECTIVE AND GLOBAL DYSTONIA REGISTRY FOR USE BY THE INTERNATIONAL DYSTONIA COMMUNITY.IN ADDITION, THE DMRF IS SERVING IN ITS FOURTEENTH YEAR WITH NO COMPENSATION AS AN ADMINISTRATIVE CENTER FOR THE DYSTONIA COALITION, WHICH is a collaboration of medical researchers and patient advocacy groups supported by the Office of Rare Diseases Research in the National Center for Advancing Translational Sciences and The National Institute of Neurological Disorders and Stroke (NINDS) at the NIH. The goal of the Coalition is to advance the pace of clinical and translational research into dystonia to find better treatments and a cure. The objectives of the Coalition are to develop a fuller understanding of the many different features of dystonia and how they change over the years, to develop validated diagnostic strategies and rating tools for diagnosis and monitoring patients in clinical trials, to establish a natural history study biorepository where samples of blood and other materials can be stored and distributed for research, to catalyze clinical trials for promising new treatments and to promote education and awareness. THE DMRF WAS RESPONSIBLE FOR MANAGING $763,342 RESTRICTED FOR DYSTONIA COALITION WORK NOT INCLUDED IN THE FOUNDATION'S REPORTED ANNUAL REVENUES AND EXPENSES. THE DMRF ORGANIZED THE DYSTONIA COALITION'S ANNUAL MEETING.The DMRF partnerS with Frontiers Media to PUBLISH THE "Dystonia", the first scientific journal dedicated exclusively to the disorder. The journal bringS visibility to the growing Dystonia field and highlightS advancements in science and clinical practice. THE DMRF HELD AN IN-PERSON EDITORIAL MEETING IN JUNE 2023.By gathering together a select group of researchers exploring this important issue, the DMRF'S GOAL IS to establish connections between great scientific minds to better understand the mechanisms of dystonia.

Program: membership and supportplanning and supporting virtual patient symposia and virtual community forums attended by patients and their families which provide information about current Research and treatment options and an opportunity to develop personal networks and reduce isolation for those affected by Dystonia; andcreating virtual educational forums for dystonia-affected persons and their families; and promoting support resources to aid in disease management and quality of life such as a network of support groups and an electronic social network, including bulletin boards and telephone communications with individuals affected by Dystonia and other interested parties; anddeveloping 58 support groups and forums in communities across the country as an enduring resource for those affected by Dystonia and their families; providing ongoing leadership training and networking for volunteer support leaders by creating electronic forums for leaders to share experiences and provide peer support, providing webinar training and ongoing support for support leaders, and distributing a support group manual which provides valuble information and resources for effective support leadership (137 support group meetings were held);andproviding meeting management support such as email and postal notifications and a web-based calendar about upcoming support group meetings including supplying Dystonia brochures to all support groups.

Program: awareness and educationpublishing the "Dystonia dialogue" news magazine three times a year under the direction of the volunteer editorial board and distributing it via mail or electronic copy to over 50,000 patients, families, and interested persons to inform them of the latest Research and Foundation activities; andcreating and disseminating over twenty-six Dystonia related educational resources as well as promoting and distributing a children's book about Dystonia and cd/dvds to requesting individuals to educate them about the various forms of Dystonia and treatment options; andhosting a comprehensive website, webinars, and electronic social networks such as the online Dystonia bulletin board, twitter, facebook, and youtube educational resources postings to provide easy access to information for affected persons and their families; andcreating and disseminating a monthly electronic newsletter and periodic email alerts to share current information on Dystonia related events, news, and activities; andcreating and disseminating a Dystonia awareness program designed to empower people to share their personal stories and increase the public's awareness of Dystonia; andparticipating in advocacy to promote awareness amongst lawmakers on topics important to Dystonia patients. See schedule o, part iii 4d for details.