This study is testing an experimental drug to help individuals with Duchenne Muscular Dystrophy. This drug might help improve dystrophin production in the muscles. Some people in the study will get the drug and some will get a placebo. This study will help us know if the drug is safe, helpful, and can be tolerated in the body.
- Male Only
- Under 18 years old
- Volunteers with special conditions
- In Person
Who can participate?
Gender: Male Only
Age: Under 18 years old
Volunteers: Volunteers with special conditions
Location: In Person
- Male children and adolescents who are 4-16 years old.
- Those who have already been diagnosed with a certain gene mutation in the dystrophin gene of their DNA.
- Those who are already taking a regular dose of glucocorticoids--such as prednisone, prednisolone, deflazacort--for at least 12 weeks.
- Those with a low level of blood that leaves the left ventricle of their heart each time their heart pumps. This is measured with an echocardiogram or a cardiac MRI.
- Those with congestive heart failure that is not under control.
- Those who have had major surgery within 12 weeks before the study, or who might have a major surgery during the study.
- Those who need ventilator assistance to breathe during the day.
Will I be paid for my time?