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Lentivirus-mediated gene therapy corrects ribosomal biogenesis and shows promise for Diamond Blackfan anemia.
Giménez Y, Palacios M, Sánchez-Domínguez R, Zorbas C, Peral J, Puzik A, Ugalde L, Alberquilla O, Villanueva M, Río P, Gálvez E, Da Costa L, Strullu M, Catala A, Ruiz-Llobet A, Segovia JC, Sevilla J, Strahm B, Niemeyer CM, Beléndez C, Leblanc T, Lafontaine DL, Bueren J, Navarro S. Giménez Y, et al. Among authors: niemeyer cm. JCI Insight. 2024 May 22;9(10):e171650. doi: 10.1172/jci.insight.171650. JCI Insight. 2024. PMID: 38775150 Free PMC article.
Diagnosis, treatment, and surveillance of Diamond-Blackfan anaemia syndrome: international consensus statement.
Wlodarski MW, Vlachos A, Farrar JE, Da Costa LM, Kattamis A, Dianzani I, Belendez C, Unal S, Tamary H, Pasauliene R, Pospisilova D, de la Fuente J, Iskander D, Wolfe L, Liu JM, Shimamura A, Albrecht K, Lausen B, Bechensteen AG, Tedgard U, Puzik A, Quarello P, Ramenghi U, Bartels M, Hengartner H, Farah RA, Al Saleh M, Hamidieh AA, Yang W, Ito E, Kook H, Ovsyannikova G, Kager L, Gleizes PE, Dalle JH, Strahm B, Niemeyer CM, Lipton JM, Leblanc TM; international Diamond-Blackfan anaemia syndrome guideline panel. Wlodarski MW, et al. Among authors: niemeyer cm. Lancet Haematol. 2024 May;11(5):e368-e382. doi: 10.1016/S2352-3026(24)00063-2. Lancet Haematol. 2024. PMID: 38697731 Review.
Variable Clinical Courses of Varicella Zoster Virus Infection-related or Vaccination-related Bone Marrow Failure.
Toskov V, Cseh A, Claviez A, Drexler B, Rotari N, Schwarz-Furlan S, Braun M, Bader P, Lang P, Beier R, Erdlenbruch B, Führer M, Erlacher M, Niemeyer CM, Strahm B, Yoshimi A. Toskov V, et al. Among authors: niemeyer cm. J Pediatr Hematol Oncol. 2024 Apr 23. doi: 10.1097/MPH.0000000000002866. Online ahead of print. J Pediatr Hematol Oncol. 2024. PMID: 38652069
Comparison of outcomes of immunosuppressive therapy with rabbit versus horse antithymocyte globulin and cyclosporine a in children with acquired severe aplastic anemia.
Yoshimi A, Noellke P, Starý J, Kállay K, Smith O, Locatelli F, Buechner J, Bodova I, Sevilla J, Schmugge M, Bierings M, Masmas T, Dworzak M, Labarque V, Pawelec K, Jahnukainen K, Polychronopoulou S, Kjollerstrom P, Kavcic M, Erlacher M, Niemeyer CM, Strahm B. Yoshimi A, et al. Among authors: niemeyer cm. Am J Hematol. 2024 Apr 15. doi: 10.1002/ajh.27334. Online ahead of print. Am J Hematol. 2024. PMID: 38622880 No abstract available.
505 results