welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!
completed

Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

key information

study id #: NCT01540409

condition: Duchenne Muscular Dystrophy (DMD)

status: completed

purpose:

The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully completed the 28 week eteplirsen study: Study 4658-us-201. This study will also evaluate the correlation between biomarkers for DMD and the clinical status of participating DMD subjects.

intervention: AVI-4658 (Eteplirsen)

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT01540409

last updated: August 01, 2019