Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)

Exclusion Criteria

• •1. Patient has had an acute illness within 4 weeks prior to the first dose of IP;
• •2. Patient has evidence of symptomatic cardiomyopathy (New York Heart Association Class III or higher);
• •3. Patient requires ventilation support while awake during the day;
• •4. Patient has an allergy or hypersensitivity to IP or any of its constituents;
• •5. Patient has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator;
• •6. Patient has a previous or ongoing medical condition, medical history, physical findings, or laboratory abnormalities that could affect patient 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;
• •7. Patient has had surgery within 3 months prior to the first anticipated administration of IP or has known plans to have surgery during the Treatment Period;
• •8. Patient has positive test results for hepatitis B antigen, hepatitis C antibody, or human immunodeficiency virus antibody at Screening;
• •9. Patient has been diagnosed with asthma that requires chronic treatment with a long-acting beta agonist;
• •10. Patient has relevant history of or current drug or alcohol abuse or use of any tobacco/marijuana products by smoking or vaping within 3 months prior to treatment with IP;
• •11. Patient is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of IP or within 5 times the half-life of a medication, whichever is longer;
• •12. Patient has taken any gene therapy;
• •13. Patient is currently taking any other exon skipping agent or has taken any other exon skipping agent within 3 months prior to the first dose of IP;
• •14. Patient has hydronephrosis, hydroureter, renal or urinary tract calculi, or ureteral stenosis by renal ultrasound;
• •15. Patient was previously enrolled in an interventional study of viltolarsen.