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The usefulness of determining plasma and tissue concentrations of phosphorodiamidate morpholino oligonucleotides to estimate their efficacy in Duchenne muscular dystrophy patients

Shunji Imai, Naoki Watanabe, Yuichiro Tone, Rei Mitamura, Jumpei Mori, Tsubasa Kameyama, Tetsuhiro Yamada and Kazutomi Kusano
Drug Metabolism and Disposition July 11, 2024, DMD-AR-2024-001806; DOI: https://doi.org/10.1124/dmd.124.001806
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Relationship between exposure and efficacy in PMOs

Shunji Imai, Naoki Watanabe, Yuichiro Tone, Rei Mitamura, Jumpei Mori, Tsubasa Kameyama, Tetsuhiro Yamada and Kazutomi Kusano
Drug Metabolism and Disposition July 11, 2024, DMD-AR-2024-001806; DOI: https://doi.org/10.1124/dmd.124.001806
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