Duchenne Outcomes Research Interchange Data Enrichment Through EHR Extraction

What this Trial Is About

This research aims to collect and combine long-term electronic health record (EHR) data and patient-reported information from individuals with dystrophinopathy, including Duchenne muscular dystrophy, Becker muscular dystrophy, and symptomatic female carriers. By gathering these data from select clinics across the United States, the study seeks to gain a fuller understanding of these conditions to help researchers and clinicians develop treatments more quickly and improve standards of care, with the goal of enhancing patient function, quality of life, and survival. The study gathers fully identifiable retrospective EHR data for core clinical elements going back up to ten years and retrospective clinical notes going back one year from the date of consent. It also collects prospective core clinical data and notes moving forward. Data collected aligns with national clinical data standards and is securely stored. Participants may be receiving various disease-modifying therapies, including FDA-approved treatments or those in clinical trials, while their data is observed and collected. Participants provide consent for their EHR data to be connected with registry information, and some may need caregiver assistance to participate. Researchers will track outcomes such as muscle weakness progression, heart and lung function, and bone health through annual clinical data and imaging over an anticipated average of 20 years. Participation is voluntary, and patients may withdraw consent at any time. The study provides ongoing monitoring through secure data collection to support better care and research.

Duchenne Electronic Health Record Study