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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: yu h. 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 Free article. Clinical Trial.
AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.
Rangarajan S, Walsh L, Lester W, Perry D, Madan B, Laffan M, Yu H, Vettermann C, Pierce GF, Wong WY, Pasi KJ. Rangarajan S, et al. Among authors: yu h. N Engl J Med. 2017 Dec 28;377(26):2519-2530. doi: 10.1056/NEJMoa1708483. Epub 2017 Dec 9. N Engl J Med. 2017. PMID: 29224506 Free article. Clinical Trial.
Valoctocogene roxaparvovec gene transfer in participants with HIV.
Ragni MV, Majerus E, Fong S, Yates B, Scheeler S, Razon L, Yu H, Reddy DB, Robinson TM. Ragni MV, et al. Among authors: yu h. Blood Adv. 2023 Apr 25;7(8):1525-1530. doi: 10.1182/bloodadvances.2022008948. Blood Adv. 2023. PMID: 36696462 Free PMC article. No abstract available.
Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.
Mahlangu J, Kaczmarek R, von Drygalski A, Shapiro S, Chou SC, Ozelo MC, Kenet G, Peyvandi F, Wang M, Madan B, Key NS, Laffan M, Dunn AL, Mason J, Quon DV, Symington E, Leavitt AD, Oldenburg J, Chambost H, Reding MT, Jayaram K, Yu H, Mahajan R, Chavele KM, Reddy DB, Henshaw J, Robinson TM, Wong WY, Pipe SW; GENEr8-1 Trial Group. Mahlangu J, et al. Among authors: yu h. N Engl J Med. 2023 Feb 23;388(8):694-705. doi: 10.1056/NEJMoa2211075. N Engl J Med. 2023. PMID: 36812433 Clinical Trial.
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: yu h. J Thromb Haemost. 2024 Jul;22(7):1880-1893. doi: 10.1016/j.jtha.2024.04.001. Epub 2024 Apr 12. J Thromb Haemost. 2024. PMID: 38614387 Free article. Clinical Trial.
Clinical immunogenicity outcomes from GENEr8-1, a phase 3 study of valoctocogene roxaparvovec, an AAV5-vectored gene therapy for hemophilia A.
Long BR, Robinson TM, Day JRS, Yu H, Lau K, Imtiaz U, Patton KS, de Hart G, Henshaw J, Agarwal S, Vettermann C, Zoog SJ, Gupta S. Long BR, et al. Among authors: yu h. Mol Ther. 2024 Jul 3;32(7):2052-2063. doi: 10.1016/j.ymthe.2024.05.033. Epub 2024 May 24. Mol Ther. 2024. PMID: 38796703 Free article. Clinical Trial.
Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial.
Leavitt AD, Mahlangu J, Raheja P, Symington E, Quon DV, Giermasz A, López Fernández MF, Kenet G, Lowe G, Key NS, Millar CM, Pipe SW, Madan B, Chou SC, Klamroth R, Mason J, Chambost H, Peyvandi F, Majerus E, Pepperell D, Rivat C, Yu H, Robinson TM, Ozelo MC. Leavitt AD, et al. Among authors: yu h. Res Pract Thromb Haemost. 2024 Oct 30;8(8):102615. doi: 10.1016/j.rpth.2024.102615. eCollection 2024 Nov. Res Pract Thromb Haemost. 2024. PMID: 39687929 Free PMC article.
Acute Ischemic Stroke Care Quality Improvement.
Zhu J, Kim KR, Yu H, Gupta A, Mushlin AI, Kamel H, Pandya A. Zhu J, et al. Among authors: yu h. JAMA Neurol. 2025 Jan 13. doi: 10.1001/jamaneurol.2024.4604. Online ahead of print. JAMA Neurol. 2025. PMID: 39804662
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