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An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients with Advanced Stage Duchenne Muscular Dystrophy (Protocol Number 4658-204)
This research study is being done to find out if eteplirsen can help people with advanced Duchenne Muscular Dystrophy (DMD). A predecessor trial has been IRB approved (IRB00040305). DMD is caused by a mutation in the gene that makes dystrophin which is important for protecting muscles from stress and damage during activity.
This research study will test whether eteplirsen works to improve muscle function in people with DMD who have deletions that may be corrected by skipping exon 51, and to find out if eteplirsen is safe to take without causing too many side effects.
Eteplirsen is designed to “skip” the exon 51 of the dystrophin gene. For people who have deletions in certain parts of the dystrophin gene, skipping exon 51 might potentially allow the body to produce a shortened, but still working, form of the dystrophin protein.
This research study will test whether eteplirsen works to improve muscle function in people with advanced DMD who have deletions that may be corrected by skipping exon 51.
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