The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation

Exclusion Criteria

• •Chronic systemic fungal or viral infections
• •An acute illness within 4 weeks prior to the first dose of viltolarsen
• •Symptomatic cardiomyopathy
• •Patient has a previous or ongoing medical condition, medical history, physical findings, or laboratory abnormality that could affect participant safety in the opinion of the treating physician
• •Surgery within the 3 months prior to the first anticipated administration of viltolarsen and in the opinion of the treating physician would impact weekly treatment schedule
• •Positive test results for hepatitis B antigen, hepatitis C antibody, or human immunodeficiency virus (HIV) antibody at screening
• •Currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of viltolarsen
• •Previously enrollment in any viltolarsen study.
• •Currently taking any other exon skipping agent or has taken any other exon skipping agent within 2 weeks prior to the first dose of viltolarsen (would need to be discontinued in order to be eligible)
• •Any gene therapy for DMD
• •Inadequate renal function as defined by a serum cystatin C \> 1.5 x upper limit of normal (ULN). If the value is \> 1.5 x ULN then the measurement can be repeated once. If repeat measurement is still \> 1.5 x ULN then the patient should be excluded.