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US 2023/0416741 A1Published application
Method of treating duchenne muscular dystrophy
AssigneeBiomarin Pharmaceutical Inc.
InventorsCharles A. O'Neill, Shripad S. Bhagwat, David Riley Neil, David Reid Jacoby
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Abstract
Provided herein are methods of treating or delaying the onset of Duchenne muscular dystrophy using modified antisense oligonucleotides.
Claims14
A method of treating a subject having DMD or delaying the onset of DMD in a subject, comprising administering to the subject AON1 at a dose of 0.4 mg/kg, 0.6 mg/kg, 0.8 mg/kg, 1.5 mg/kg, 3 mg/kg, 6 mg/kg, 9 mg/kg, 12 mg/kg or 18 mg/kg, wherein the AON1 is administered QW.
Dependencies are inferred from the claim text.