The Data Management and Coordinating Center (DMCC) and the Rare Diseases Clinical Research Network Consortia Principal Investigators (PIs) met in Rockville, MD for their spring meeting. Dr. Jeffrey Krischer and Consortia PIs saw this meeting as an opportunity to thank Dr. Steve Groft once more in person for his lifetime achievement in rare diseases.
The Dystonia Coalition (DC) is an international collaboration of biomedical researchers, patient advocacy groups (PAGs), and representatives from the NIH. The DC was first funded in 2009 by the Office of Rare Diseases Research at the National Center for Advancing Translational Research and the National Institute of Neurological Diseases and Stroke, during the second cycle of the Rare Disease Clinical Research Network.
The primary dystonias, where dystonia is a relatively isolated problem, have been suspected to have a genetic basis for more than 40 years. The vast majority of cases are sporadic and emerge in adults, but approximately 15% of cases have an affected family member.
The Dystonia Coalition Career Development Award has benefited my career far beyond the scope of the small project it supported. The aim of the project was to investigate transcranial magnetic stimulation and rehabilitation in focal hand dystonia. This investigation has resulted in strong pilot data and will soon be submitted for publication.
The European research community, like the North American community, has recognized the value of collaboration for advancing clinical and translational research for rare diseases such as dystonia. In recent years, many members of the European community have come together to cooperate on important initiatives.
Integral to the mission of the Dystonia Coalition (DC) are Patient Advocacy Groups (PAGs) that provide vital support, important collaborations, and help with bridging the gap between researchers and patients. Sixteen different patient advocacy groups with representation from the USA, Canada, and Europe support the Dystonia Coalition's goals of advancing the pace of clinical and translational research in the dystonias.
The Rare Diseases Clinical Research Network is an initiative of the Office of Rare Diseases Research. National Center for Advancing Translational Sciences (NCATS) funded through a collaboration between NCATS and the National Institutes of Health.