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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.
Genetic variants of VWF gene in type 2 von Willebrand disease.
Freitas SDS, Rezende SM, de Oliveira LC, Prezotti ANL, Renni MS, Corsini CA, Amorim MVA, Matosinho CGR, Carvalho MRS, Chaves DG. Freitas SDS, et al. Among authors: prezotti anl. Haemophilia. 2019 Mar;25(2):e78-e85. doi: 10.1111/hae.13714. Epub 2019 Feb 28. Haemophilia. 2019. PMID: 30817071
A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A.
Peyvandi F, Mannucci PM, Garagiola I, El-Beshlawy A, Elalfy M, Ramanan V, Eshghi P, Hanagavadi S, Varadarajan R, Karimi M, Manglani MV, Ross C, Young G, Seth T, Apte S, Nayak DM, Santagostino E, Mancuso ME, Sandoval Gonzalez AC, Mahlangu JN, Bonanad Boix S, Cerqueira M, Ewing NP, Male C, Owaidah T, Soto Arellano V, Kobrinsky NL, Majumdar S, Perez Garrido R, Sachdeva A, Simpson M, Thomas M, Zanon E, Antmen B, Kavakli K, Manco-Johnson MJ, Martinez M, Marzouka E, Mazzucconi MG, Neme D, Palomo Bravo A, Paredes Aguilera R, Prezotti A, Schmitt K, Wicklund BM, Zulfikar B, Rosendaal FR. Peyvandi F, et al. N Engl J Med. 2016 May 26;374(21):2054-64. doi: 10.1056/NEJMoa1516437. N Engl J Med. 2016. PMID: 27223147 Free article. Clinical Trial.
Immunogenicity of Current and New Therapies for Hemophilia A.
Prezotti ANL, Frade-Guanaes JO, Yamaguti-Hayakawa GG, Ozelo MC. Prezotti ANL, et al. Pharmaceuticals (Basel). 2022 Jul 23;15(8):911. doi: 10.3390/ph15080911. Pharmaceuticals (Basel). 2022. PMID: 35893734 Free PMC article. Review.