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101 results

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Page 1
Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1.
O'Mahony B, Dunn AL, Leavitt AD, Peyvandi F, Ozelo MC, Mahlangu J, Peerlinck K, Wang JD, Lowe GC, Tan CW, Giermasz A, Tran H, Khoo TL, Cockrell E, Pepperell D, Chambost H, López Fernández MF, Kazmi R, Majerus E, Skinner MW, Klamroth R, Quinn J, Yu H, Wong WY, Robinson TM, Pipe SW. O'Mahony B, et al. Among authors: lopez fernandez mf. J Thromb Haemost. 2023 Dec;21(12):3450-3462. doi: 10.1016/j.jtha.2023.08.032. Epub 2023 Sep 6. J Thromb Haemost. 2023. PMID: 37678546 Clinical Trial.
Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura.
Scully M, Antun A, Cataland SR, Coppo P, Dossier C, Biebuyck N, Hassenpflug WA, Kentouche K, Knöbl P, Kremer Hovinga JA, López-Fernández MF, Matsumoto M, Ortel TL, Windyga J, Bhattacharya I, Cronin M, Li H, Mellgård B, Patel M, Patwari P, Xiao S, Zhang P, Wang LT; cTTP Phase 3 Study Investigators. Scully M, et al. Among authors: lopez fernandez mf. N Engl J Med. 2024 May 2;390(17):1584-1596. doi: 10.1056/NEJMoa2314793. N Engl J Med. 2024. PMID: 38692292 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.
von Willebrand factor neutralizing and non-neutralizing alloantibodies in 213 subjects with type 3 von Willebrand disease enrolled in 3WINTERS-IPS.
Pagliari MT, Budde U, Baronciani L, Eshghi P, Ahmadinejad M, Badiee Z, Baghaipour MR, Benítez Hidalgo O, Biguzzi E, Bodó I, Castaman G, Goudemand J, Karimi M, Keikhaei B, Lassila R, Leebeek FWG, Lopez Fernandez MF, Marino R, Oldenburg J, Peake I, Santoro C, Schneppenheim R, Tiede A, Toogeh G, Tosetto A, Trossaert M, Yadegari H, Zetterberg EMK, Mannucci PM, Federici AB, Eikenboom J, Peyvandi F. Pagliari MT, et al. Among authors: lopez fernandez mf. J Thromb Haemost. 2023 Apr;21(4):787-799. doi: 10.1016/j.jtha.2023.01.001. Epub 2023 Jan 11. J Thromb Haemost. 2023. PMID: 36792472 Free article.
Long-Read Sequencing Identifies the First Retrotransposon Insertion and Resolves Structural Variants Causing Antithrombin Deficiency.
de la Morena-Barrio B, Stephens J, de la Morena-Barrio ME, Stefanucci L, Padilla J, Miñano A, Gleadall N, García JL, López-Fernández MF, Morange PE, Puurunen M, Undas A, Vidal F, Raymond FL, Vicente V, Ouwehand WH, Corral J, Sanchis-Juan A; NIHR BioResource. de la Morena-Barrio B, et al. Among authors: lopez fernandez mf. Thromb Haemost. 2022 Aug;122(8):1369-1378. doi: 10.1055/s-0042-1749345. Epub 2022 Jun 28. Thromb Haemost. 2022. PMID: 35764313 Free PMC article.
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.
Von Willebrand factor propeptide and pathophysiological mechanisms in European and Iranian patients with type 3 von Willebrand disease enrolled in the 3WINTERS-IPS study.
Pagliari MT, Rosendaal FR, Ahmadinejad M, Badiee Z, Baghaipour MR, Baronciani L, Benítez Hidalgo O, Bodó I, Budde U, Castaman G, Eshghi P, Goudemand J, Karimi M, Keikhaei B, Lassila R, Leebeek FWG, Lopez Fernandez MF, Mannucci PM, Marino R, Oldenburg J, Peake I, Santoro C, Schneppenheim R, Tiede A, Toogeh G, Tosetto A, Trossaert M, Yadegari H, Zetterberg EMK, Peyvandi F, Federici AB, Eikenboom J. Pagliari MT, et al. Among authors: lopez fernandez mf. J Thromb Haemost. 2022 May;20(5):1106-1114. doi: 10.1111/jth.15658. Epub 2022 Feb 22. J Thromb Haemost. 2022. PMID: 35092343 Free PMC article.
IX international curse of continuing formation in haemophilia and other congenital coagulopathies. The role of the Laboratory in coagulation disorders. Diagnosis of von Willebrand disease.
Batlle J, Pérez-Rodríguez A, Corrales I, Borràs N, Pinto JC, López-Fernández MF, Vidal F; on behalf PCM-EVW-ES Investigators Group. Batlle J, et al. Among authors: lopez fernandez mf. Blood Coagul Fibrinolysis. 2022 Jan 1;33(Suppl 1):S12-S14. doi: 10.1097/MBC.0000000000001093. Blood Coagul Fibrinolysis. 2022. PMID: 34783692
101 results