Longitudinal motor function in proximal versus distal DMD pathogenic variants
Abstract
There is considerable heterogenicity in clinical outcomes in Duchenne muscular dystrophy (DMD). The aim of this study was to assess whether dystrophin gene (DMD) pathogenic variant location influences upper or lower extremity motor function outcomes in a large prospective cohort.
Document Details
- Document Type
- Pub Defense Publication
- Publication Date
- Jul 23, 2021
- Source ID
- 10.1002/mus.27371
Entities
People
- Cinrg‐dnhs Investigators
- Heather Gordish‐dressman
- Luca Bello
- Mathula Thangarajh
Organizations
- Children's National Hospital
- National Institute of Arthritis and Musculoskeletal and Skin Diseases
- National Institute on Disability, Independent Living, and Rehabilitation Research
- National Institutes of Health
- Parent Project Muscular Dystrophy
- United States Department of Defense
- University of Padua
- Virginia Commonwealth University