At Rush University Medical Center, we are putting your safety first. For information about COVID-19, see the latest updates. Rush accepts donations to support our response effort, staff, and patients and families.

Excellence is just the beginning.


French German Italian Portuguese Russian

Nonsense Mutation Dystrophinopathy and Ataluren Treatment Study

Clinical Trial Title: 
Phase 3 efficacy and safety study of Ataluren (PTC124) in patients with nonsense mutation dystrophinopathy
Clinical Trial Protocol ID: 
Clinical Trial Investigator Name: 
Peter Heydemann, MD
Clinical Trial Protocol Description: 

Rush University Medical Center is recruiting for the Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in patients with nonsense mutation dystrophinopathy. This is an international study expecting to recruit 225 boys ages 7 to 16 with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation. As of the end of September, Peter Heydemann, MD, has enrolled two boys with another three ready to start once they soon meet eligibility requirements.

DMD is a disabling and life-threatening disorder caused by defects in the gene for dystrophin, a protein that stabilizes muscle cell membranes. Until now, the only course of treatment involved corticosteroids, which merely slows progression. In previous studies, Ataluren earned a favorable safety profile and an earlier study showed that Ataluren can restore production of the missing dystrophin.

Recruitment remains open until the study is filled. For more information, please contact Susan Rohde at (312) 942-0079.

Clinical Trial Eligibility Criteria: 

In order to participate you must meet the following criteria:

  • Are a male between the ages of 7 to 16 with a nonsense mutation form of DMD and on a current regiment of corticosteroids.

This is a partial list of eligibility requirements. 

Clinical Trial Area: 
Pediatric Neurological Disorders
Contact Phone: 
(312) 942-0079
Contact Name: 
Susan Rohde