Duchenne muscular dystrophy
Solid Biosciences Inc. focuses on neuromuscular disease research, with activity centered on Duchenne muscular dystrophy and the development of gene therapy approaches aimed at restoring microdystrophin expression in muscle tissue.
- Muscle biopsy biomarker assessment
- Microdystrophin expression
- Intravenous gene delivery
The sponsor’s clinical research interest is concentrated on treatment strategies for the genetic basis of Duchenne muscular dystrophy, with emphasis on skeletal muscle function and disease-related protein restoration.
