Natural History Studies – Neuromuscular Research Center

PITT0112: Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping.

Principal Investigator

Erik K. Henricson, Ph.D., M.P.H.


Candace Aguilar, B.A.

Study Details

Brief Summary
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."

View this study at


Inclusion Criteria:

  • Males
  • 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

Exclusion Criteria:

  • Investigator assessment of inability to comply with protocol

Recruitment Status

Currently Recruiting