At Rush University Medical Center, we are putting your safety first. For information about COVID-19, see the latest updates. Rush accepts donations to support our response effort, staff, and patients and families.
Duchenne Muscular Dystrophy Treatment Study
This study is investigating Eteplirsen, a study drug designed to skip a certain fragment of the dystrophin gene called exon 51. In people with changes (referred to as deletions) in certain parts of the dystrophin gene (including exons 45-50, 47-50, 48-50, 49-50, 50, 52, or 52-63), skipping exon 51 is believed to allow the body to produce a shortened, but still working, form of dystrophin protein. This may result in improved muscle function for people with Duchenne muscular dystrophy.
This study will examine the effects of continued treatment with Eteplirsen in children with Duchenne muscular dystrophy who have successfully completed the 28-week eteplirsen study, Study 4658-us-201.
Subjects must have successfully completed the 28-week Eteplirsen study: Study 4658-us-201.