) This application proposes funding for the 11th meeting of the International Myotonic Dystrophy Consortium (IDMC-11). IDMC-11 is scheduled to convene in San Francisco, CA from September 5th to September 9th 2017. The theme of IDMC-11 is: ?RNA Targeting Enters the Therapeutic Era?. The objective of the conference is to bring together top scientists and clinicians from around the world to discuss high-impact, interdisciplinary research on myotonic dystrophy type 1 (DM1) and DM type 2 (DM2). This meeting brings together leading basic researchers in the field 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-11 will be the participation of young investigators to co-chair sessions, present data during poster and oral presentations, and to develop collaborations with senior faculty. Funding from the NIH will be instrumental in allowing graduate students, post-doctoral fellows, and other young investigators to attend this meeting. The first day of IDMC-11 will include: registration, a keynote lecture on modifying DNA and RNA 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. Discussions on therapeutic development will be expanded upon at IDMC-11 to include two new symposiums to begin and end the scientific sessions. The opening symposium is titled, ?Therapeutic Targeting of RNA? and includes keynote seminars from leaders in academia and biotechnology to present information on antisense oligonucleotides, other small molecules, and other experimental therapies in DM1 and DM2. The closing symposium is titled, ?Biomarkers, Outcome Measures, Registries, Trial Design? with keynote presentations about clinical trial readiness in myotonic dystrophy and other neuromuscular diseases. 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-11 will focus on disease mechanisms of DM1 and DM2, animal and cell models, clinical aspects and management of disease symptoms, and therapeutic development. 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.
This application proposes funding for the 11th meeting of the International Myotonic Dystrophy Consortium (IDMC-11), which facilitates clinical and scientific discussions amongst experts in myotonic dystrophy, colleagues in pharmaceutical and regulatory agencies, members of patient advocacy groups, and patients with myotonic dystrophies and their caregivers. Young investigators are encouraged to participate at IDMC to foster their careers and to develop collaborative and translational studies with more senior colleagues. The IDMC meeting is the only conference in the world that brings together such a diverse group of attendees, including the leading experts in the field dedicated to the study, treatment, and cure of myotonic dystrophy.
