In order to develop better methods for capturing the perspective of individuals living with Duchenne muscular dystrophy (DMD) during clinical trials, researchers at the University of California Davis Neuromuscular Research Center are seeking participants for an online survey to monitor movement and daily living abilities. The Duchenne Muscular Dystrophy Life-Time Mobility Scale (DMD-LMS) is a set of questions that ask about how easy or hard it is for individuals with DMD to complete specific movements and activities in their day-to-day life. Once validated, this survey could serve as a critical new outcome measure for clinical trials.
The goal of the survey is to provide additional information that can help researchers better describe ways that new therapies might benefit patients at home and in the community. Evaluation of strength and functional abilities for individuals with DMD is usually performed at clinical visits by doctors or physical therapists.
Although those tests work well for tracking changes in ability, they do not always describe how a person with DMD functions outside the clinic or research laboratory. Additionally, the DMD-LMS can be completed much more frequently than clinic visits and tests and can be done from anywhere, including in the home. Parent/guardian- and self-reported questionnaires about community mobility and day-to-day physical ability, when used along with clinical strength and function testing, can provide valuable additional information about how changes in strength affect people with DMD in day-to-day life.
Support by participants will ensure the use of the DMD-LMS as a patient-reported outcome measurement in future DMD clinical trials.
About the survey
Participants will spend approximately 30 minutes answering questions about how easy or hard a movement or task would be if the person with DMD had to complete it within the past seven days without help. Each question represents a specific type of body movement that is done during specific day-to-day tasks (such as walking in the community, or standing up from a chair or drinking from a glass). Taken together, how a person with DMD (or their parent/guardian) responds to each of the questions describes how strong they are and how well they are able to perform day-to-day tasks.
Participating in the Survey
UC Davis is recruiting up to 1,000 individuals with DMD ages 5 years and older and/or their parents/guardians to participate in the online survey project. Participants will answer the questionnaire in three stages: at enrollment, one month later and one year later.
Parents of children from 5-17 years will answer questions on behalf of their children. Adolescents 11-17 years old with DMD will answer the same questions as their parents or guardians. Adults with DMD will answer the questions without their parents/guardians.
For more information and to access the study link, click here.