An Open-Label, Multi-Center, 48-Week Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
A study for male adolecents with muscular dystrophy using study drug Eteplirsen
Sponsor: Sarepta Therapeutics
Enrolling: Male and Female Patients
IRB Number: AAAN5951
U.S. Govt. ID: NCT02255552
Contact: Jonathan Marra: 212-342-3022 / jdm2132@cumc.columbia.edu
Additional Study Information: The purpose of this study is to find out if eteplirsen can help people with the muscle disorder called Duchenne Muscular Dystrophy (DMD) and find out if eteplirsen is safe to take without causing too many side effects. Eteplirsen is an investigational (experimental) drug and can only be used in research studies. DMD is caused by a mutation (a change) in the gene (the part of cells that contain the instructions which tell our bodies how to grow and work) that makes dystrophin (a protein). Dystrophin is important for protecting muscles from stress and damage during activity. If a person has DMD, their body is not able to make enough working dystrophin to protect their muscles.
This study is closed
Investigator
Jacinda Sampson, MD
Do You Qualify?
Has your son been diagnosed with Duchenne Muscular Dystrophy? Yes No
Has your son had previous treatment with drisapersen within the last 6 months? Yes No
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You may be eligible for this study

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For more information, please contact:
Jonathan Marra
jdm2132@cumc.columbia.edu
212-342-3022