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Current Research Studies

A phase 3, randomized, double - blind, placebo - controlled efficacy and safety study of ataluren in patients with nonsense mutation Duchenne Muscular Dystrophy and open - label extension.

DMD_AtalurenRCT41

Clinicaltrials.gov ID: NCT03179631

What is the goal of the study?

This is a randomized controlled trial of ambulatory boys ages 5 years and older with Duchenne Muscular Dystrophy (DMD) due to a nonsense mutation in the dystrophin gene, with boys receiving either placebo or ataluren for 72 weeks, then receiving ataluren for 72 weeks. Boys have follow up visits during the study including physical therapy testing, respiratory and cardiac monitoring, and physical exams.

Who can participate in the study?

Please contact the study team listed below to learn more.

Study Team:

PI (Study Doctor): Seth Perlman
Study Team Member: Jacqueline Lee-Eng, jacqueline.lee-eng@seattlechildrens.org

Seattle Children’s complies with applicable federal and other civil rights laws and does not discriminate, exclude people or treat them differently based on race, color, religion (creed), sex, gender identity or expression, sexual orientation, national origin (ancestry), age, disability, or any other status protected by applicable federal, state or local law. Financial assistance for medically necessary services is based on family income and hospital resources and is provided to children under age 21 whose primary residence is in Washington, Alaska, Montana or Idaho.