US2022403385A1PendingUtilityA1

Compositions for treating muscular dystrophy

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Assignee: SAREPTA THERAPEUTICS INCPriority: Mar 15, 2013Filed: Jan 3, 2022Published: Dec 22, 2022
Est. expiryMar 15, 2033(~6.6 yrs left)· nominal 20-yr term from priority
Inventors:Edward M. Kaye
A61P 21/00C12N 15/113A61P 21/02A61P 1/00C12N 2310/33A61K 31/7125A61K 9/0019C12N 2320/30
75
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Claims

Abstract

Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described.

Claims

exact text as granted — not AI-modified
1 . A method for treating Duchenne muscular dystrophy in a patient in need thereof comprising administering to the patient a dose of eteplirsen of about 30 mg/kg once a week.

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