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19 results

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Page 1
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.
Ozelo MC, Mahlangu J, Pasi KJ, Giermasz A, Leavitt AD, Laffan M, Symington E, Quon DV, Wang JD, Peerlinck K, Pipe SW, Madan B, Key NS, Pierce GF, O'Mahony B, Kaczmarek R, Henshaw J, Lawal A, Jayaram K, Huang M, Yang X, Wong WY, Kim B; GENEr8-1 Trial Group. Ozelo MC, et al. N Engl J Med. 2022 Mar 17;386(11):1013-1025. doi: 10.1056/NEJMoa2113708. N Engl J Med. 2022. PMID: 35294811 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.
Prediction of inhibitor development in previously untreated and minimally treated children with severe and moderately severe hemophilia A using a machine-learning network.
Jardim LL, Schieber TA, Santana MP, Cerqueira MH, Lorenzato CS, Franco VKB, Zuccherato LW, da Silva Santos BA, Chaves DG, Ravetti MG, Rezende SM. Jardim LL, et al. Among authors: lorenzato cs. J Thromb Haemost. 2024 Sep;22(9):2426-2437. doi: 10.1016/j.jtha.2024.05.017. Epub 2024 May 27. J Thromb Haemost. 2024. PMID: 38810700 Free article.
Large deletions and small insertions and deletions in the factor VIII gene predict unfavorable immune tolerance induction outcome in people with severe hemophilia A and high-responding inhibitors.
Zuccherato LW, Souza RP, Camelo RM, Dias MM, Jardim LL, Santana MAP, Oliveira AG, Lorenzato CS, Cerqueira MH, Franco VKB, Ribeiro RA, Etto LY, Roberti MDRF, Callado FMA, de Cerqueira MAF, Pinto ISS, Garcia AA, Anegawa TH, Neves DCF, Tan DM, Tou RP, Chaves DG, van der Bom J, Rezende SM; HEMFIL Study and the Brazilian Immune Tolerance (BrazIT) Study. Zuccherato LW, et al. Among authors: lorenzato cs. Thromb Res. 2024 Oct;242:109115. doi: 10.1016/j.thromres.2024.109115. Epub 2024 Aug 14. Thromb Res. 2024. PMID: 39186847
Prediction of the chance of successful immune tolerance induction in persons with severe hemophilia A and inhibitors: a clinical prediction model.
Oomen I, Abdi A, Camelo RM, Callado FMRA, Carvalho LEM, Calcaterra IL, Carcao M, Castaman G, Eikenboom JCJ, Fischer K, Franco VKB, Heymans MW, Leebeek FWG, Lillicrap D, Lorenzato CS, Mancuso ME, Matino D, Di Minno MND, Mohseny AB, Oldenburg J, Rezende SM, Rivard GE, Rydz N, Schols SEM, Voorberg J, Fijnvandraat K, Gouw SC; International Genetic and clinical determinants for the outcome of immune tolerance induction study group. Oomen I, et al. Among authors: lorenzato cs. Res Pract Thromb Haemost. 2024 Oct 3;8(7):102580. doi: 10.1016/j.rpth.2024.102580. eCollection 2024 Oct. Res Pract Thromb Haemost. 2024. PMID: 39558913 Free PMC article.
Changing recombinant factor VIII to plasma-derived factor VIII during immune tolerance induction.
Dias MM, Mesquita Camelo R, de Magalhães LP, Lemos Jardim L, Gonçalves de Oliveira A, Ribeiro RA, Karla Brognoli Franco V, Callado FMRA, Lorenzato CS, Rezende SM; Brazilian Immune Tolerance (BrazIT) Study group. Dias MM, et al. Among authors: lorenzato cs. Pediatr Hematol Oncol. 2024;41(1):74-80. doi: 10.1080/08880018.2023.2182853. Epub 2023 Feb 24. Pediatr Hematol Oncol. 2024. PMID: 36825651 No abstract available.
Multimodal molecular imaging of atherosclerosis: Nanoparticles functionalized with scFv fragments of an anti-αIIbβ3 antibody.
Larivière M, Lorenzato CS, Adumeau L, Bonnet S, Hémadou A, Jacobin-Valat MJ, Noubhani A, Santarelli X, Minder L, Di Primo C, Sanchez S, Mornet S, Laroche-Traineau J, Clofent-Sanchez G. Larivière M, et al. Among authors: lorenzato cs. Nanomedicine. 2019 Nov;22:102082. doi: 10.1016/j.nano.2019.102082. Epub 2019 Aug 9. Nanomedicine. 2019. PMID: 31404651
Time between inhibitor detection and start of immune tolerance induction: Association with outcome in the BrazIT study.
Camelo RM, Dias MM, Caram-Deelder C, Gouw S, de Magalhães LP, Zuccherato LW, Jardim LL, de Oliveira AG, de Albuquerque Ribeiro R, Franco VKB, do Rosário Ferraz Roberti M, de Araújo Callado FMR, Etto LY, de Cerqueira MAF, Cerqueira MH, Lorenzato CS, de Souza IS, Serafim ÉSS, Garcia AA, Anegawa TH, Neves DCF, Tan DM, van der Bom J, Rezende SM; Brazilian Immune Tolerance (BrazIT) Study group. Camelo RM, et al. Among authors: lorenzato cs. J Thromb Haemost. 2022 Nov;20(11):2526-2537. doi: 10.1111/jth.15878. Epub 2022 Sep 26. J Thromb Haemost. 2022. PMID: 36102352 Free article.
19 results