BMD Natural History Study
Natural History Studies – Neuromuscular Research Center
PITT0112: Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping.
This study is looking at patients with BMD to develop a better understanding of how certain changes in the dystrophin gene affect the disease progression, physical development, mental development, and quality of life in patients with BMD.
There are no interventions for this study. We call these studies "Natural History Studies."
- Age 4 and above
- Diagnosis of BMD confirmed by the central CINRG genetic counselor with a positive DMD deletion for one of the following: exons 45-47, exons 45-48, exons 45-51, exons 45-53, or exons 48-51 where the boundaries of the mutations are confirmed
- Investigator assessment of inability to comply with protocol