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Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.
Birch SM, Lawlor MW, Conlon TJ, Guo LJ, Crudele JM, Hawkins EC, Nghiem PP, Ahn M, Meng H, Beatka MJ, Fickau BA, Prieto JC, Styner MA, Struharik MJ, Shanks C, Brown KJ, Golebiowski D, Bettis AK, Balog-Alvarez CJ, Clement N, Coleman KE, Corti M, Pan X, Hauschka SD, Gonzalez JP, Morris CA, Schneider JS, Duan D, Chamberlain JS, Byrne BJ, Kornegay JN. Birch SM, et al. Among authors: nghiem pp. Sci Transl Med. 2023 Jan 4;15(677):eabo1815. doi: 10.1126/scitranslmed.abo1815. Epub 2023 Jan 4. Sci Transl Med. 2023. PMID: 36599002 Free PMC article.
Use of the six-minute walk test to characterize golden retriever muscular dystrophy.
Acosta AR, Van Wie E, Stoughton WB, Bettis AK, Barnett HH, LaBrie NR, Balog-Alvarez CJ, Nghiem PP, Cummings KJ, Kornegay JN. Acosta AR, et al. Among authors: nghiem pp. Neuromuscul Disord. 2016 Dec;26(12):865-872. doi: 10.1016/j.nmd.2016.09.024. Epub 2016 Oct 3. Neuromuscul Disord. 2016. PMID: 27818009
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