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Springer Nature1
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Pharmaceutical Research1
Publication Year
20211
Bryan Mastis results 1 · Newest (Page 1/1, per page 25)
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Pharmacokinetic/Pharmacodynamic Modeling of a Cell-Penetrating Peptide Phosphorodiamidate Morpholino Oligomer in mdx MiceNARA Subscribed
Pharmaceutical Research · 2021 · Vol. 38 · Issue 10 · Springer
Marie Claire Mukashyaka; Chia-Ling Wu; Kristin Ha; Jianbo Zhang; Jenna Wood; Samantha Foley; Bryan Mastis; Nino Jungels; Huadong Sun; Mohammad Shadid; Shawn Harriman; John R. Hadcock
Purpose Peptide-conjugated phosphorodiamidate morpholino oligomers (PPMOs) have shown promise in treating Duchenne muscular dystrophy (DMD). We evaluated a semi-mechanistic pharmacokinetic (PK) and pharmacodynamic (PD) model to capture the relationship between plasma and muscle tissue exposure/response in mdx mice treated by mouse surrogate PPMO. Methods A single or repeated (every 4 weeks for 20 weeks) intravenous PPMO dose w...
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