Researchers at the University of Rochester in New York seek patients diagnosed with Duchenne muscular dystrophy (DMD) who are interested in helping to develop disease-specific patient-reported outcome measures for future clinical trials. This study will help to determine the most critical symptoms of children, young adults, and adult patients with DMD, and as a result, it will allow researchers to measure small but clinically significant therapeutic gains during future clinical trials. Throughout this study, researchers will conduct qualitative patient interviews that will shed light on the most important symptoms of patients with DMD. Ultimately, it will be used to create a questionnaire that will measure disease burden within the DMD population.

About the survey

The first part of the study involves a qualitative interview over the phone. Using open-ended questions, researchers will ask participants to identify key symptoms of DMD that have an impact on their quality of life. There are two groups of interest:

• Ages 11-17
• Ages 18 and above

For minors, interviews will last up to 30 minutes, or as long as participants are able to participate. Minors would need a parent to provide verbal permission to the study coordinator, and would need to offer their own verbal consent. For adults, interviews will last around 60 minutes and will require their verbal consent as well. All interviews will be recorded and transcribed for later reference, and this data will be kept in a secure location. The goal is to complete approximately 15 minor interviews and 15 adult interviews.

Participants

Participants must be 11 years or older, English speaking, and diagnosed with DMD based on a report from their physician or MDA’s registry. No travel is required, as interviews occur over the phone.

To learn more and to inquire about participation, contact Christine Zizzi at 585-276-7772 or Christine.zizzi@chet.rochester.edu.