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Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents.
Landrum Peay H, Fischer R, Tzeng JP, Hesterlee SE, Morris C, Strong Martin A, Rensch C, Smith E, Ricotti V, Beaverson K, Wand H, Mansfield C. Landrum Peay H, et al. Among authors: morris c. PLoS One. 2019 May 1;14(5):e0213649. doi: 10.1371/journal.pone.0213649. eCollection 2019. PLoS One. 2019. PMID: 31042754 Free PMC article.
A mouse anti-myostatin antibody increases muscle mass and improves muscle strength and contractility in the mdx mouse model of Duchenne muscular dystrophy and its humanized equivalent, domagrozumab (PF-06252616), increases muscle volume in cynomolgus monkeys.
St Andre M, Johnson M, Bansal PN, Wellen J, Robertson A, Opsahl A, Burch PM, Bialek P, Morris C, Owens J. St Andre M, et al. Among authors: morris c. Skelet Muscle. 2017 Nov 9;7(1):25. doi: 10.1186/s13395-017-0141-y. Skelet Muscle. 2017. PMID: 29121992 Free PMC article.
Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients.
Paquin RS, Fischer R, Mansfield C, Mange B, Beaverson K, Ganot A, Martin AS, Morris C, Rensch C, Ricotti V, Russo LJ, Sadosky A, Smith EC, Peay HL. Paquin RS, et al. Among authors: morris c. Orphanet J Rare Dis. 2019 May 9;14(1):102. doi: 10.1186/s13023-019-1069-6. Orphanet J Rare Dis. 2019. PMID: 31072340 Free PMC article.
Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy.
Peay HL, Fischer R, Mange B, Paquin RS, Smith EC, Sadosky A, Russo L, Ricotti V, Rensch C, Morris C, Martin AS, Ganot A, Beaverson K, Mansfield C. Peay HL, et al. Among authors: morris c. Mol Genet Genomic Med. 2021 May;9(5):e1664. doi: 10.1002/mgg3.1664. Epub 2021 Mar 23. Mol Genet Genomic Med. 2021. PMID: 33755338 Free PMC article.
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: morris ca. 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.
Anti-latent TGFβ binding protein 4 antibody improves muscle function and reduces muscle fibrosis in muscular dystrophy.
Demonbreun AR, Fallon KS, Oosterbaan CC, Vaught LA, Reiser NL, Bogdanovic E, Velez MP, Salamone IM, Page PGT, Hadhazy M, Quattrocelli M, Barefield DY, Wood LD, Gonzalez JP, Morris C, McNally EM. Demonbreun AR, et al. Among authors: morris c. Sci Transl Med. 2021 Sep 8;13(610):eabf0376. doi: 10.1126/scitranslmed.abf0376. Epub 2021 Sep 8. Sci Transl Med. 2021. PMID: 34516828 Free PMC article.
4,302 results