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198 results

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Page 1
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.
Pipe SW, Leebeek FWG, Recht M, Key NS, Castaman G, Miesbach W, Lattimore S, Peerlinck K, Van der Valk P, Coppens M, Kampmann P, Meijer K, O'Connell N, Pasi KJ, Hart DP, Kazmi R, Astermark J, Hermans CRJR, Klamroth R, Lemons R, Visweshwar N, von Drygalski A, Young G, Crary SE, Escobar M, Gomez E, Kruse-Jarres R, Quon DV, Symington E, Wang M, Wheeler AP, Gut R, Liu YP, Dolmetsch RE, Cooper DL, Li Y, Goldstein B, Monahan PE. Pipe SW, et al. Among authors: wheeler ap. N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644. N Engl J Med. 2023. PMID: 36812434 Clinical Trial.
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. N Engl J Med. 2023 Feb 23;388(8):694-705. doi: 10.1056/NEJMoa2211075. N Engl J Med. 2023. PMID: 36812433 Clinical Trial.
Treatment practices and response in kaposiform hemangioendothelioma: A multicenter cohort study.
Borst AJ, Eng W, Griffin M, Ricci KW, Engel E, Adams DM, Dayneka J, Cohen-Cutler SJ, Andreoli SM, Wu MD, Wheeler AP, Heym KM, Crary SE, Nakano TA, Schulte RR, Setty BA, McLean TW, Pahl KS, Intzes S, Pateva I, Teitelbaum M, Zong Z, Li Y, Jeng MR. Borst AJ, et al. Among authors: wheeler ap. Pediatr Blood Cancer. 2024 Mar;71(3):e30779. doi: 10.1002/pbc.30779. Epub 2023 Dec 10. Pediatr Blood Cancer. 2024. PMID: 38073018
Acute pulmonary injury in hematology patients supported with pathogen-reduced and conventional platelet components.
Wheeler AP, Snyder EL, Refaai M, Cohn CS, Poisson J, Fontaine M, Sehl M, Nooka AK, Uhl L, Spinella PC, Fenelus M, Liles D, Coyle T, Becker J, Jeng M, Gehrie EA, Spencer BR, Young P, Johnson A, O'Brien JJ, Schiller GJ, Roback JD, Malynn E, Jackups R, Avecilla ST, Liu K, Bentow S, Varrone J, Benjamin RJ, Corash LM. Wheeler AP, et al. Blood Adv. 2024 May 14;8(9):2290-2299. doi: 10.1182/bloodadvances.2023012425. Blood Adv. 2024. PMID: 38447116 Free PMC article.
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial.
Coppens M, Pipe SW, Miesbach W, Astermark J, Recht M, van der Valk P, Ewenstein B, Pinachyan K, Galante N, Le Quellec S, Monahan PE, Leebeek FWG; HOPE-B Investigators. Coppens M, et al. Lancet Haematol. 2024 Apr;11(4):e265-e275. doi: 10.1016/S2352-3026(24)00006-1. Epub 2024 Mar 1. Lancet Haematol. 2024. PMID: 38437857 Clinical Trial.
Author Correction: Toll-like receptor 9 protects non-immune cells from stress by modulating mitochondrial ATP synthesis through the inhibition of SERCA2.
Shintani Y, Drexler HC, Kioka H, Terracciano CM, Coppen SR, Imamura H, Akao M, Nakai J, Wheeler AP, Higo S, Nakayama H, Takashima S, Yashiro K, Suzuki K. Shintani Y, et al. Among authors: wheeler ap. EMBO Rep. 2024 Apr;25(4):2145. doi: 10.1038/s44319-024-00100-1. EMBO Rep. 2024. PMID: 38429580 Free PMC article.
198 results