Participate in Research at CU Anschutz

Child

Inclusion Criteria 1. Patient's parent(s) or legal guardian(s) has (have) provided written informed consent; patients will be asked to give written or verbal assent according to local requirements. 2. Patient has a confirmed diagnosis of DMD 3. Patient is >+4 years and <15 years of age at time of first infusion in the study. 4. Patient is able to walk independently without assistive devices. 5. Patient is able to complete the TTRW and TTCLIMB 6. Patient is able to complete the TTSTAND without assistance in <7 seconds 7. Patient and parent(s)/guardian(s) are willing and able to comply with scheduled study visits, study drug administration plan, and study procedures. 8. Patient must be on a stable dose of glucocorticoid (GC) for at least 3 months prior to first dose of study drug and is expected to remain on the stable dose of GC treatment for the duration of the study. 9. Patient, if sexually active with a partner of childbearing potential, must use a male condom during and for 3 months following completion of study drug administration. The sexual partner must also use a medically acceptable form of contraception (eg, female oral contraceptives) during this timeframe. Exclusion Criteria 1. Patient has current or history of chronic systemic fungal or viral infections. 2. Patient has had an acute illness within 4 weeks prior to the first dose of study drug based on the investigator's judgment/discretion. 3. Patient has a QTc >450 msec on standard 12-lead electrocardiogram (ECG) or, if patient has bundle branch block, QTc >480 msec. 4. Patient has a left ventricular ejection fraction (LVEF) of <40% or fractional shortening <25%. 5. Patient has evidence of symptomatic cardiomyopathy. Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary. 6. Patient has an allergy or hypersensitivity to the study medication or to any of its constituents (eg, sucrose). 7. Patient has severe behavioral or cognitive problems that preclude participation in the study in the opinion of the investigator. 8. Patient has previous or ongoing medical condition, medical history, physical findings, or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed, or impair the assessment of study results, in the opinion of the investigator. 9. Patient is currently taking another investigational drug or has taken another investigational drug within 3 months prior to the first dose of study drug. 10. Patient has had surgery within the 3 months prior to the first anticipated administration of study medication, or surgery is planned for anytime during the duration of the study. 11. Patient has taken any gene therapy. 12. Patient has positive test results for hepatitis B antigen, hepatitis C antibody, or human immunodeficiency virus (HIV) antibody at screening. 13. Patient is currently taking anabolic steroids (eg, oxendolone, oxandrolone), or products containing resveratrol or adenosine triphosphate, or has taken such within 3 months prior to the first dose of study drug. 14. Patient has hydronephrosis, hydroureter, renal calculi, or ureteral stenosis by medical history or abdominal ultrasound. 15. Patient is unable or unwilling to undergo MRI or patient has contraindications to MRI Procedure. Note: Applicable only for patients ≥7 years old. 16. Patient is unable or unwilling to pause use of anticoagulants, non-steroidal anti-inflammatory agents, or chronic antiplatelet agents for 3 days prior to muscle biopsy. 17. Patient is unable to perform muscle biopsy due to platelet count <120,000 platelets per microliter, history of uncontrolled bleeding disorder, or advanced wasting of muscle to be biopsied. 18. Patient has clinically significant illness such as abnormal renal, hepatic, hematologic, or immunologic illness, or any other reason as judged by the investigator. 19. Patient is judged by the investigator to be inappropriInclusion Criteria 1. Patient's parent(s) or legal guardian(s) has (have) provided written informed consent; patients will be asked to give written or verbal assent according to local requirements. 2. Patient has a confirmed diagnosis of DMD 3. Patient is >+4 years and <15 years of age at time of first infusion in the study. 4. Patient is able to walk independently without assistive devices. 5. Patient is able to complete the TTRW and TTCLIMB 6. Patient is able to complete the TTSTAND without assistance in <7 seconds 7. Patient and parent(s)/guardian(s) are willing and able to comply with scheduled study visits, study drug administration plan, and study procedures. 8. Patient must be on a stable dose of glucocorticoid (GC) for at least 3 months prior to first dose of study drug and is expected to remain on the stable dose of GC treatment for the duration of the study. 9. Patient, if sexually active with a partner of childbearing potential, must use a male condom during and for 3 months following completion of study drug administration. The sexual partner must also use a medically acceptable form of contraception (eg, female oral contraceptives) during this timeframe. Exclusion Criteria 1. Patient has current or history of chronic systemic fungal or viral infections. 2. Patient has had an acute illness within 4 weeks prior to the first dose of study drug based on the investigator's judgment/discretion. 3. Patient has a QTc >450 msec on standard 12-lead electrocardiogram (ECG) or, if patient has bundle branch block, QTc >480 msec. 4. Patient has a left ventricular ejection fraction (LVEF) of <40% or fractional shortening <25%. 5. Patient has evidence of symptomatic cardiomyopathy. Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary. 6. Patient has an allergy or hypersensitivity to the study medication or to any of its constituents (eg, sucrose).