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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
2005 1
2006 2
2008 2
2009 1
2010 2
2011 5
2012 6
2013 6
2014 3
2015 1
2016 3
2018 5
2019 6
2020 11
2021 13
2022 9
2023 10
2024 1

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73 results

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Page 1
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.
Achieving the unimaginable: Health equity in haemophilia.
Skinner MW, Nugent D, Wilton P, O'Mahony B, Dolan G, O'Hara J, Berntorp E. Skinner MW, et al. Haemophilia. 2020 Jan;26(1):17-24. doi: 10.1111/hae.13862. Epub 2019 Nov 13. Haemophilia. 2020. PMID: 31724316 Free PMC article. Review.
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.
World Federation of Hemophilia Gene Therapy Registry.
Konkle BA, Coffin D, Pierce GF, Clark C, George L, Iorio A, Mahlangu J, Naccache M, O'Mahony B, Peyvandi F, Pipe S, Quartel A, Sawyer EK, Skinner MW, Tortella B, Watson C, Winburn I; Members of the WFH Gene Therapy Registery Steering Committee. Konkle BA, et al. Among authors: skinner mw. Haemophilia. 2020 Jul;26(4):563-564. doi: 10.1111/hae.14015. Epub 2020 May 27. Haemophilia. 2020. PMID: 32462720 Free PMC article. No abstract available.
Achieving access to haemophilia care in low-income and lower-middle-income countries: expanded Humanitarian Aid Program of the World Federation of Hemophilia after 5 years.
Pierce GF, Adediran M, Diop S, Dunn AL, El Ekiaby M, Kaczmarek R, Konkle BA, Pipe SW, Skinner MW, Valentino LA, Robinson F, Ampartzidis G, Martin J, Haffar A. Pierce GF, et al. Among authors: skinner mw. Lancet Haematol. 2022 Sep;9(9):e689-e697. doi: 10.1016/S2352-3026(22)00209-5. Lancet Haematol. 2022. PMID: 36055333 Review.
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
Eliminating Panglossian thinking in development of AAV therapeutics.
Kaczmarek R, Pierce GF, Noone D, O'Mahony B, Page D, Skinner MW. Kaczmarek R, et al. Among authors: skinner mw. Mol Ther. 2021 Dec 1;29(12):3325-3327. doi: 10.1016/j.ymthe.2021.10.025. Epub 2021 Nov 10. Mol Ther. 2021. PMID: 34758292 Free PMC article. No abstract available.
73 results