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2005 1
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2013 6
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2020 12
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2023 10
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74 results

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Page 1
Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1.
O'Mahony B, Dunn AL, Leavitt AD, Peyvandi F, Ozelo MC, Mahlangu J, Peerlinck K, Wang JD, Lowe GC, Tan CW, Giermasz A, Tran H, Khoo TL, Cockrell E, Pepperell D, Chambost H, López Fernández MF, Kazmi R, Majerus E, Skinner MW, Klamroth R, Quinn J, Yu H, Wong WY, Robinson TM, Pipe SW. O'Mahony B, et al. Among authors: skinner mw. J Thromb Haemost. 2023 Dec;21(12):3450-3462. doi: 10.1016/j.jtha.2023.08.032. Epub 2023 Sep 6. J Thromb Haemost. 2023. PMID: 37678546 Clinical Trial.
Hemophilia gene therapy: first, do no harm.
Valentino LA, Kaczmarek R, Pierce GF, Noone D, O'Mahony B, Page D, Rotellini D, Skinner MW. Valentino LA, et al. Among authors: skinner mw. J Thromb Haemost. 2023 Sep;21(9):2354-2361. doi: 10.1016/j.jtha.2023.06.016. Epub 2023 Jun 21. J Thromb Haemost. 2023. PMID: 37353081 Free article. Review.
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science.
Byams VR, Baker JR, Bailey C, Connell NT, Creary MS, Curtis RG, Dinno A, Guelcher CJ, Kim M, Kulkarni R, Lattimore S, Norris KL, Ramirez L, Skinner MW, Symington S, Tobase P, Vázquez E, Warren BB, Wheat E, Buckner TW. Byams VR, et al. Among authors: skinner mw. Expert Rev Hematol. 2023 Mar;16(sup1):87-106. doi: 10.1080/17474086.2023.2183836. Expert Rev Hematol. 2023. PMID: 36920863
Data sharing to advance gene-targeted therapies in rare diseases.
Lekstrom-Himes J, Augustine EF, Brower A, Defay T, Finkel RS, McGuire AL, Skinner MW, Yu TW. Lekstrom-Himes J, et al. Among authors: skinner mw. Am J Med Genet C Semin Med Genet. 2023 Mar;193(1):87-98. doi: 10.1002/ajmg.c.32028. Epub 2023 Jan 3. Am J Med Genet C Semin Med Genet. 2023. PMID: 36594517
74 results