Due to a measles case in the community, please call before coming to Seattle Children’s if you or your child has potential measles symptoms.

Masking and Visitation Changes: Due to high rates of respiratory illnesses in our community, we’ve made changes to our masking and visitation guidelines .

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.

RS_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, [email protected]