Collaboration with DuchenneConnect Patient-Powered Research Network

Duchenne and Becker Muscular Dystrophy causes progressive muscle weakness and early death, and there are no FDA-approved therapies. Engaging patients and their caregivers and families to understand their priorities, needs, and preferences leads to more active participation in disease management and clinical research. This collaboration will include identification of a DBMD specialist, pediatric neurologist Dr. Katherine Mathews, to coordinate the efforts of DCN PPRN and GPC CDRN; developing processes for data exchange; engagement about data transparency policies for collaboration with PPRN's; and development of a decision aid tool for patients around clinical trial participation. By bringing together patient and clinician entered data, we can empower patients to contribute vital information about their health, function and quality of life.

Other Information:

• Principal Investigator: Holly Peay, MS; John Hopkins Bloomberg School of Public Health