Clinical Study Alert: Natural History Studies of Individuals with DMD and Their Caregivers

Researchers at the Center for Health + Technology at the University of Rochester are seeking individuals with Duchenne muscular dystrophy (DMD) and their adult caregivers to participate in observational REDCap surveys, DMD-Health Index (DMD-HI) and DMD Caregiver Reported-Health Index (DMDCR-HI) respectively. The findings of these surveys will help to determine how DMD disease burden changes over time and identify demographic and clinical factors associated with faster or slower progression of disease.

These two observational studies last for 24 months. Individuals with DMD who are enrolled in the DMD-HI study will be asked to complete a demographics questionnaire, the Duchene Muscular Dystrophy-Health Index (DMD-HI), the PedsQL DMD Module (patient-reported), and an instrument preference survey at baseline, 6 months, 12 months, 18 months, and 24 months. Similarly, caregivers enrolled in the DMDCR-HI study will be asked to complete a demographics questionnaire, the Duchene Muscular Dystrophy Caregiver Reported-Health Index (DMDCR-HI), the Peds QL DMD Module (caregiver-reported) or the non-disease specific PedsQL Proxy survey, and an instrument preference survey at baseline, 6 months, 12 months, 18 months, and 24 months.

To be eligible for the DMD-HI research study, individuals must meet the following inclusion criteria:

• Be an individual 11 years old and older
• Be an individual with a diagnosis of DMD
• Speak English
• Have the capacity to give informed consent

To be eligible for the DMDCR-HI research study, individuals must meet the following inclusion criteria:

• Be 18 years of age and older
• Care for an individual between 0-21 years of age
• Caring for an individual with a diagnosis of DMD
• Speak English
• Have the capacity to give informed consent

Participants may self-enroll to the DMD-HI study by clicking on https://redcap.link/DMD-HI_Longitudinal or the DMDCR-HI study by clicking on https://redcap.link/DMDCR-HI_Longitudinal. To learn more about the study or inquire about participation, contact the study coordinator Jennifer Weinstein by email at: jennifer.weinstein@chet.rochester.edu.