Report of the first Outcome Measures in Myotonic Dystrophy type 1 (OMMYD-1) international workshop. Clearwater, Florida, November 30, 2011.

摘要

For this first workshop, a total of 31 attendees (clinicians and researchers working in myotonic dystrophy type 1 (DM1)) from France, Canada, USA, the UK, Germany, Sweden and Italy met in Clearwater, Florida on November 30, 2011. The overall-goal of the Outcome Measures in myotonic dystrophy type 1 (OMMYD-1) workshop is to select outcome measures (OM) that can be used in RCTs for the development of new therapeutics for DM1. Before the meeting, a few necessary steps were completed by all participants to ensure better efficiency. First, a literature review was made to find information about OM used in DM1 and other diseases. Then, documents concerning these OM and references relative to the applicability and metrological properties of these OM were sent to participants. They were invited to provide comments before the meeting to improve workshop planning. The OMMYD-1 conference was based on the methodology developed by the OMERACT group in the rheumatology field for the development and selection of outcome measures [1] and it was divided into five Special Interest Groups (SIG): (1) Cognitive functions; (2) Quality of life; (3) Upper and lower extremity functions; (4) Muscle testing and training; and (5) Development of a new disease severity index for DM1 (DSI-DM1).