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187 results

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Page 1
Aberrant activation of TCL1A promotes stem cell expansion in clonal haematopoiesis.
Weinstock JS, Gopakumar J, Burugula BB, Uddin MM, Jahn N, Belk JA, Bouzid H, Daniel B, Miao Z, Ly N, Mack TM, Luna SE, Prothro KP, Mitchell SR, Laurie CA, Broome JG, Taylor KD, Guo X, Sinner MF, von Falkenhausen AS, Kääb S, Shuldiner AR, O'Connell JR, Lewis JP, Boerwinkle E, Barnes KC, Chami N, Kenny EE, Loos RJF, Fornage M, Hou L, Lloyd-Jones DM, Redline S, Cade BE, Psaty BM, Bis JC, Brody JA, Silverman EK, Yun JH, Qiao D, Palmer ND, Freedman BI, Bowden DW, Cho MH, DeMeo DL, Vasan RS, Yanek LR, Becker LC, Kardia SLR, Peyser PA, He J, Rienstra M, Van der Harst P, Kaplan R, Heckbert SR, Smith NL, Wiggins KL, Arnett DK, Irvin MR, Tiwari H, Cutler MJ, Knight S, Muhlestein JB, Correa A, Raffield LM, Gao Y, de Andrade M, Rotter JI, Rich SS, Tracy RP, Konkle BA, Johnsen JM, Wheeler MM, Smith JG, Melander O, Nilsson PM, Custer BS, Duggirala R, Curran JE, Blangero J, McGarvey S, Williams LK, Xiao S, Yang M, Gu CC, Chen YI, Lee WJ, Marcus GM, Kane JP, Pullinger CR, Shoemaker MB, Darbar D, Roden DM, Albert C, Kooperberg C, Zhou Y, Manson JE, Desai P, Johnson AD, Mathias RA; NHLBI Trans-Omics for Precision Medicine (TOPMed) Consortium; Blackwell TW, Abecasis GR, Smith AV, Kang HM, Sat… See abstract for full author list ➔ Weinstock JS, et al. Among authors: konkle ba. Nature. 2023 Apr;616(7958):755-763. doi: 10.1038/s41586-023-05806-1. Epub 2023 Apr 12. Nature. 2023. PMID: 37046083 Free PMC article.
Management of pregnant women who have bleeding disorders.
James AH, Pacheco LD, Konkle BA. James AH, et al. Among authors: konkle ba. Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):229-236. doi: 10.1182/hematology.2023000475. Hematology Am Soc Hematol Educ Program. 2023. PMID: 38066866
Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A.
von Drygalski A, Chowdary P, Kulkarni R, Susen S, Konkle BA, Oldenburg J, Matino D, Klamroth R, Weyand AC, Jimenez-Yuste V, Nogami K, Poloskey S, Winding B, Willemze A, Knobe K; XTEND-1 Trial Group. von Drygalski A, et al. Among authors: konkle ba. N Engl J Med. 2023 Jan 26;388(4):310-318. doi: 10.1056/NEJMoa2209226. N Engl J Med. 2023. PMID: 36720133 Clinical Trial.
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.
Landmark endorsement of a global registry: The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP), publicly endorses World Federation of Hemophilia Gene Therapy Registry as global standard.
Konkle BA, Peyvandi F, Coffin D, Naccache M, Youttananukorn T, Pierce GF; WFH Gene Therapy Registry Scientific Advisory Board. Konkle BA, et al. Haemophilia. 2024 Jan;30(1):232-235. doi: 10.1111/hae.14912. Epub 2023 Dec 18. Haemophilia. 2024. PMID: 38111095 No abstract available.
A global comparative field study to evaluate the factor VIII activity of efanesoctocog alfa by one-stage clotting and chromogenic substrate assays at clinical haemostasis laboratories.
Pipe S, Sadeghi-Khomami A, Konkle BA, Kitchen S, Negrier C, Liu M, Santagostino E, Willemze A, Abad-Franch L, Knobe K, Seth Chhabra E. Pipe S, et al. Among authors: konkle ba. Haemophilia. 2024 Jan;30(1):214-223. doi: 10.1111/hae.14831. Epub 2023 Oct 30. Haemophilia. 2024. PMID: 37902390
Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study.
Leavitt AD, Konkle BA, Stine KC, Visweshwar N, Harrington TJ, Giermasz A, Arkin S, Fang A, Plonski F, Yver A, Ganne F, Agathon D, Resa MLA, Tseng LJ, Di Russo G, Cockroft BM, Cao L, Rupon J. Leavitt AD, et al. Among authors: konkle ba. Blood. 2024 Feb 29;143(9):796-806. doi: 10.1182/blood.2022018971. Blood. 2024. PMID: 37871576 Free PMC article. Clinical Trial.
187 results