This application proposes an international conference entitled: "Economic Considerations in the Development of New Treatment for Neuromuscular Diseases". In addition sessions will: (1) provide updates on the most exciting developments in research in neuromuscular diseases to presentations on the many economic issues important in bringing new therapies to patients;(2) updates on ongoing and planned neuromuscular disease trials;and (3) sessions on how to begin to participate in multicenter studies. The conference brings together translational and clinical scientists from academia, foundations, and pharma in a collegial forum. This proposal has been developed by co-chairs working with the leadership of the Muscle Study Group. The conference is open to attendance from the Muscle Study Group, the British Myology Society and other groups and by individuals not currently affiliated with a study group. Priority is given to investigators submitting research for presentation. The conference's scientific emphasis will be on key cross-cutting themes common to multiple neuromuscular disorders that have clinical importance and translational potential. The conference will be held in an environment that will bring senior investigators, representatives of pharma, National Institutes of Health program staff, and foundation and patient advocacy representatives together with junior/trainee investigators. The conference provides perspective for trainees and junior faculty in the strategies for developing novel treatments for neuromuscular diseases. The setting and the meeting format foster collaboration among established investigators and between senior and new/junior investigators. It is expected that the conference will continue to lead to new collaborations among attendees. All attendees will be invited to submit their work and to participate in interactive poster sessions;the most interesting submissions will be the basis of 2 sessions for platform presentation. Trainees will be encouraged and supported to attend and to present their own work. Abstracts of all presentations will be published and presentations will be made available on the Muscle Study Group website. The conference is designed to encourage and assist residents, trainees and junior faculty to pursue a career in experimental therapeutics.
This is an application for funds for an international conference which will bring together scientists from academia, foundations, federal agencies and industry to discuss research strategies and to prepare for collaborative studies of new treatments for neuromuscular diseases. Part of this year's meeting will have a focus on economic issues important to bringing new treatments to patients. A major goal of the meeting is to attract and mentor new junior scientists and trainees in the scientific underpinning and methods of clinical and translational neuroscience.