DOSING OF MUSCLE TARGETING COMPLEXES FOR TREATING DYSTROPHINOPATHIES
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United States of America
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Jan 16, 2025
app pub date -
Sep 15, 2022
filing date -
Sep 15, 2022
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Abstract
Aspects of the disclosure relate to methods of promoting expression or activity of a dystrophin protein and/or methods of treating DMD in a subject. In some embodiments, the methods comprise administering to the subject a composition comprising complexes (e.g., muscle targeting complexes) comprising a phosphorodiamidate morpholino oligomer (e.g., useful for targeting DMD) covalently linked to an antibody (e.g., anti-TfR1 antibody).
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