This R13 application seeks funding for the 12th meeting of the International Myotonic Dystrophy Consortium (IDMC-12). Since 1997, the biennial IDMC meeting has served as the preeminent international forum for research on myotonic dystrophy. The next meeting in the series, IDMC-12, is scheduled to convene in Gothenburg, Sweden from June 10?14, 2019. The objective of the conference is to bring together top scientists, clinicians, and drug developers from around the world to discuss high-impact, interdisciplinary research on myotonic dystrophy type 1 (DM1) and DM type 2 (DM2). The conference is attended by leading basic and translational researchers in the field, together with clinicians and clinical investigators representing a broad range of specialties including neurology, cardiology, anesthesiology, clinical trials, pathology, genetics, and molecular biology. Their interactions and discussions stimulate new approaches to research and clinical care and provide opportunities to develop and strengthen collaborative studies. A major focus at IDMC-12 will be placed on the participation of young investigators to co-chair sessions, present data during poster and oral presentations, and develop collaborations. Funding from the NIH will be instrumental in allowing graduate students and post-doctoral fellows to attend this meeting. The first day of IDMC-12 will include: registration, a keynote lecture by Dr. David Nelson, co-discoverer of the CGG expansion in fragile X syndrome, and a welcoming reception. The subsequent three days of the meeting will be devoted to scientific sessions in which researchers and clinicians present peer-reviewed selected abstracts on basic, clinical and translational research topics. As compared to previous meetings in the series, discussions on therapeutic development will be expanded upon at IDMC-12, to include results from recent clinical trials, efforts to develop biomarkers and clinical endpoints to assess therapeutic response, and progress in developing small molecule, oligonucleotide, and viral vectors treatments for DM1 and DM2. The scientific sessions begin at 8:00 AM and conclude between 5:30 to 7:30 PM each day. There will be three formal poster sessions. The sessions at IDMC-12 will focus on pathogenetic mechanisms, cell and animal models, drug development and delivery, clinical research on biomarkers and outcome measures, therapeutic trials, clinical guidelines and rehabilitation, quality of life and disease burden, and genetic diagnosis and counseling. On the final day, there will be patient-oriented sessions to promote discussion among patients, care-givers, representatives of patient advocacy groups, clinicians, and investigators. The final afternoon will begin with highlights from the previous sessions, followed by advocacy group presentations, and a session for questions from patients and family members.

Public Health Relevance

The 12th biennial conference of the International Myotonic Dystrophy Consortium (IDMC-12) will focus on the entire process of therapeutic development from bench to bedside. The meeting is designed to facilitate discussions and collaborations amongst a broad range of experts, including those from academia, industry, government, patient advocacy groups, and patients with myotonic dystrophy (DM) and their caregivers. The IDMC conference will continue to serve as the only international meeting that comprehensively involves all stakeholders in the DM field for therapeutic development.

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Conference (R13)
Project #
1R13NS113630-01
Application #
9837036
Study Section
Special Emphasis Panel (ZNS1)
Program Officer
Nuckolls, Glen H
Project Start
2019-07-01
Project End
2020-06-30
Budget Start
2019-07-01
Budget End
2020-06-30
Support Year
1
Fiscal Year
2019
Total Cost
Indirect Cost
Name
University of Rochester
Department
Neurology
Type
School of Medicine & Dentistry
DUNS #
041294109
City
Rochester
State
NY
Country
United States
Zip Code
14627