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Page 1
A Molecular Revolution in the Treatment of Hemophilia.
Butterfield JSS, Hege KM, Herzog RW, Kaczmarek R. Butterfield JSS, et al. Among authors: kaczmarek r. Mol Ther. 2020 Apr 8;28(4):997-1015. doi: 10.1016/j.ymthe.2019.11.006. Epub 2019 Nov 13. Mol Ther. 2020. PMID: 31843450 Free PMC article. Review.
Curing Hemophilia: Repeated Treatments versus a One-Off Fix.
Li N, Kaczmarek R. Li N, et al. Among authors: kaczmarek r. Mol Ther. 2020 May 6;28(5):1229-1230. doi: 10.1016/j.ymthe.2020.04.012. Epub 2020 Apr 17. Mol Ther. 2020. PMID: 32304671 Free PMC article. No abstract available.
B cell-activating factor modulates the factor VIII immune response in hemophilia A.
Doshi BS, Rana J, Castaman G, Shaheen MA, Kaczmarek R, Butterfield JS, Meeks SL, Leissinger C, Biswas M, Arruda VR. Doshi BS, et al. Among authors: kaczmarek r. J Clin Invest. 2021 Apr 15;131(8):e142906. doi: 10.1172/JCI142906. J Clin Invest. 2021. PMID: 33651716 Free PMC article. Clinical Trial.
Eliminating Panglossian thinking in development of AAV therapeutics.
Kaczmarek R, Pierce GF, Noone D, O'Mahony B, Page D, Skinner MW. Kaczmarek R, et al. Mol Ther. 2021 Dec 1;29(12):3325-3327. doi: 10.1016/j.ymthe.2021.10.025. Epub 2021 Nov 10. Mol Ther. 2021. PMID: 34758292 Free PMC article. No abstract available.
Treatment-induced hemophilic thrombosis?
Kaczmarek R, Herzog RW. Kaczmarek R, et al. Mol Ther. 2022 Feb 2;30(2):505-506. doi: 10.1016/j.ymthe.2022.01.015. Epub 2022 Jan 20. Mol Ther. 2022. PMID: 35063080 Free PMC article. No abstract available.
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. Among authors: kaczmarek r. N Engl J Med. 2022 Mar 17;386(11):1013-1025. doi: 10.1056/NEJMoa2113708. N Engl J Med. 2022. PMID: 35294811 Clinical Trial.
Safety and efficacy of emicizumab and other novel agents in newborns and infants.
Pierce GF, Hart DP, Kaczmarek R; Committee on Coagulation Products Safety Supply, Access (CPSSA) of the World Federation of Hemophilia (WFH). Pierce GF, et al. Among authors: kaczmarek r. Haemophilia. 2019 Sep;25(5):e334-e335. doi: 10.1111/hae.13822. Epub 2019 Jul 30. Haemophilia. 2019. PMID: 31361382 No abstract available.
Towards a global multidisciplinary consensus framework on haemophilia gene therapy: Report of the 2nd World Federation of Haemophilia Gene Therapy Round Table.
Pierce GF, Pasi KJ, Coffin D, Kaczmarek R, Lillicrap D, Mahlangu J, Rottellini D, Sannié T, Srivastava A, VandenDriessche T, Weill A; Members of the WFH Gene Therapy Round Table Program Organizing Committee. Pierce GF, et al. Among authors: kaczmarek r. Haemophilia. 2020 May;26(3):443-449. doi: 10.1111/hae.13971. Epub 2020 Mar 23. Haemophilia. 2020. PMID: 32202382
Management of COVID-19-associated coagulopathy in persons with haemophilia.
Pipe SW, Kaczmarek R, Srivastava A, Pierce GF, Makris M, Hermans C; Interim Guidance; Coagulation Products Safety, Supply and Access (CPSSA) Committee of the World Federation of Hemophilia. Pipe SW, et al. Among authors: kaczmarek r. Haemophilia. 2021 Jan;27(1):41-48. doi: 10.1111/hae.14191. Epub 2020 Nov 20. Haemophilia. 2021. PMID: 33216448 Free PMC article.
174 results