Online Self-Report Data for Duchenne Muscular Dystrophy Confirms Natural History and Can Be Used to Assess for Therapeutic Benefits

CONCLUSION
The data presented here establish DuchenneConnect as a useful framework for further growth in online patient self-report data that is valuable for therapeutic interpretations regarding DMD, and support the more general use of online self-report data for therapeutic interpretation in rare disease. In five years, DuchenneConnect has accumulated the largest DMD cohort with age at full time wheelchair use and treatment information published to date, likely because of the low barrier to participation. Further, with many current registrants passing major milestones, like loss of ambulation, and the ongoing inclusion of new registrants, DuchenneConnect is poised to provide an ongoing and important longitudinal resource for observation of current DMD clinical care to support observations from clinical networks, clinical trial networks, or other natural history data. Thus, continued collection of data from web-registered participants promises to provide important natural history, clinical practice, and treatment response data for DMD. This study represents a successful, mutually beneficial collaboration between academic investigators and a patient self-report registry supported by an advocacy organization. Consistent with recent moves to embrace patient generated data, this study and the ongoing collaboration are informing the efficient use of patient-report data to describe patient outcomes and guide care provision.

Source: PLOS Currents Muscular Dystrophy - October 17, 2014 Category: Neurology Authors: richardwang Source Type: research