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101 results

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The translational gap for gene therapies in low- and middle-income countries.
Doxzen KW, Adair JE, Fonseca Bazzo YM, Bukini D, Cornetta K, Dalal V, Guerino-Cunha RL, Hongeng S, Jotwani G, Kityo-Mutuluuza C, Lakshmanan K, Mahlangu J, Makani J, Mathews V, Ozelo MC, Rangarajan S, Scholefield J, Batista Silva Júnior J, McCune JM. Doxzen KW, et al. Among authors: ozelo mc. Sci Transl Med. 2024 May 8;16(746):eadn1902. doi: 10.1126/scitranslmed.adn1902. Epub 2024 May 8. Sci Transl Med. 2024. PMID: 38718130 Review.
Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A.
Madan B, Ozelo MC, Raheja P, Symington E, Quon DV, Leavitt AD, Pipe SW, Lowe G, Kenet G, Reding MT, Mason J, Wang M, von Drygalski A, Klamroth R, Shapiro S, Chambost H, Dunn AL, Oldenburg J, Chou SC, Peyvandi F, Millar CM, Osmond D, Yu H, Dashiell-Aje E, Robinson TM, Mahlangu J. Madan B, et al. Among authors: ozelo mc. J Thromb Haemost. 2024 Apr 12:S1538-7836(24)00184-3. doi: 10.1016/j.jtha.2024.04.001. Online ahead of print. J Thromb Haemost. 2024. PMID: 38614387 Free article.
Current and emerging gene therapies for haemophilia A and B.
Kaczmarek R, Miesbach W, Ozelo MC, Chowdary P. Kaczmarek R, et al. Among authors: ozelo mc. Haemophilia. 2024 Apr;30 Suppl 3:12-20. doi: 10.1111/hae.14984. Epub 2024 Mar 25. Haemophilia. 2024. PMID: 38528615
Comparative transcriptomic analysis of circulating endothelial cells in sickle cell stroke.
de Castro JNP, da Silva Costa SM, Camargo ACL, Ito MT, de Souza BB, de Haidar E Bertozzo V, Rodrigues TAR, Lanaro C, de Albuquerque DM, Saez RC, Saad STO, Ozelo MC, Cendes F, Costa FF, de Melo MB. de Castro JNP, et al. Among authors: ozelo mc. Ann Hematol. 2024 Apr;103(4):1167-1179. doi: 10.1007/s00277-024-05655-6. Epub 2024 Feb 22. Ann Hematol. 2024. PMID: 38386032
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: ozelo mc. 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.
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