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178 results

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The patient's view on rare disease trial design - a qualitative study.
Gaasterland CMW, van der Weide MCJ, du Prie-Olthof MJ, Donk M, Kaatee MM, Kaczmarek R, Lavery C, Leeson-Beevers K, O'Neill N, Timmis O, van Nederveen V, Vroom E, van der Lee JH. Gaasterland CMW, et al. Among authors: kaczmarek r. Orphanet J Rare Dis. 2019 Feb 7;14(1):31. doi: 10.1186/s13023-019-1002-z. Orphanet J Rare Dis. 2019. PMID: 30732630 Free PMC article.
The POWER-tool: Recommendations for involving patient representatives in choosing relevant outcome measures during rare disease clinical trial design.
Gaasterland CMW, Jansen-van der Weide MC, Vroom E, Leeson-Beevers K, Kaatee M, Kaczmarek R, Bartels B, van der Pol WL, Roes KCB, van der Lee JH. Gaasterland CMW, et al. Among authors: kaczmarek r. Health Policy. 2018 Dec;122(12):1287-1294. doi: 10.1016/j.healthpol.2018.09.011. Epub 2018 Sep 21. Health Policy. 2018. PMID: 30274934 Free article.
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.
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.
Gene therapy - are we ready now?
Kaczmarek R. Kaczmarek R. Haemophilia. 2022 May;28 Suppl 4(Suppl 4):35-43. doi: 10.1111/hae.14530. Haemophilia. 2022. PMID: 35521736 Free PMC article.
A single point mutation in the gene encoding Gb3/CD77 synthase causes a rare inherited polyagglutination syndrome.
Suchanowska A, Kaczmarek R, Duk M, Lukasiewicz J, Smolarek D, Majorczyk E, Jaskiewicz E, Laskowska A, Wasniowska K, Grodecka M, Lisowska E, Czerwinski M. Suchanowska A, et al. Among authors: kaczmarek r. J Biol Chem. 2012 Nov 2;287(45):38220-30. doi: 10.1074/jbc.M112.408286. Epub 2012 Sep 10. J Biol Chem. 2012. PMID: 22965229 Free PMC article.
178 results