Valoctocogene roxaparvovec gene therapy for hemophilia A MC Ozelo, J Mahlangu, KJ Pasi, A Giermasz, AD Leavitt, M Laffan, ... New England Journal of Medicine 386 (11), 1013-1025, 2022 | 323 | 2022 |
Two-year outcomes of valoctocogene roxaparvovec therapy for hemophilia A J Mahlangu, R Kaczmarek, A Von Drygalski, S Shapiro, SC Chou, ... New England Journal of Medicine 388 (8), 694-705, 2023 | 147 | 2023 |
A molecular revolution in the treatment of hemophilia JSS Butterfield, KM Hege, RW Herzog, R Kaczmarek Molecular Therapy 28 (4), 997-1015, 2020 | 93 | 2020 |
Erythrocyte glycophorins as receptors for Plasmodium merozoites E Jaskiewicz, M Jodłowska, R Kaczmarek, A Zerka Parasites & vectors 12, 1-11, 2019 | 92 | 2019 |
The patient’s view on rare disease trial design–a qualitative study CMW Gaasterland, MCJ van der Weide, MJ du Prie–Olthof, M Donk, ... Orphanet journal of rare diseases 14, 1-9, 2019 | 64 | 2019 |
How glycosylation affects glycosylation: the role of N-glycans in glycosyltransferase activity K Mikolajczyk, R Kaczmarek, M Czerwinski Glycobiology 30 (12), 941-969, 2020 | 61 | 2020 |
A single point mutation in the gene encoding Gb3/CD77 synthase causes a rare inherited polyagglutination syndrome A Suchanowska, R Kaczmarek, M Duk, J Lukasiewicz, D Smolarek, ... Journal of Biological Chemistry 287 (45), 38220-38230, 2012 | 53 | 2012 |
P1PK, GLOB, and FORS blood group systems and GLOB collection: biochemical and clinical aspects. Do we understand it all yet? R Kaczmarek, A Buczkowska, K Mikołajewicz, H Krotkiewski, ... Transfusion medicine reviews 28 (3), 126-136, 2014 | 51 | 2014 |
Achieving access to haemophilia care in low-income and lower-middle-income countries: expanded Humanitarian Aid Program of the World Federation of Hemophilia after 5 years GF Pierce, M Adediran, S Diop, AL Dunn, M El Ekiaby, R Kaczmarek, ... The Lancet Haematology 9 (9), e689-e697, 2022 | 47 | 2022 |
Human Gb3/CD77 synthase reveals specificity toward two or four different acceptors depending on amino acid at position 211, creating Pk, P1 and NOR blood group antigens R Kaczmarek, M Duk, K Szymczak, E Korchagina, J Tyborowska, ... Biochemical and biophysical research communications 470 (1), 168-174, 2016 | 31 | 2016 |
The Gerbich blood group system: old knowledge, new importance E Jaskiewicz, T Peyrard, R Kaczmarek, A Zerka, M Jodlowska, ... Transfusion medicine reviews 32 (2), 111-116, 2018 | 30 | 2018 |
Gene therapy to cure haemophilia: Is robust scientific inquiry the missing factor? GF Pierce, R Kaczmarek, D Noone, B O’Mahony, D Page, MW Skinner Haemophilia 26 (6), 931-933, 2020 | 29 | 2020 |
The baculovirus-expressed binding region of Plasmodium falciparum EBA-140 ligand and its glycophorin C binding specificity J Rydzak, R Kaczmarek, M Czerwinski, J Lukasiewicz, J Tyborowska, ... PLoS One 10 (1), e0115437, 2015 | 28 | 2015 |
First conditional marketing authorization approval in the European Union for hemophilia “A” gene therapy T VandenDriessche, SW Pipe, GF Pierce, R Kaczmarek Molecular Therapy 30 (11), 3335-3336, 2022 | 25 | 2022 |
B cell–activating factor modulates the factor VIII immune response in hemophilia A BS Doshi, J Rana, G Castaman, MA Shaheen, R Kaczmarek, ... The Journal of Clinical Investigation 131 (8), 2021 | 24 | 2021 |
Management of COVID‐19‐associated coagulopathy in persons with haemophilia SW Pipe, R Kaczmarek, A Srivastava, GF Pierce, M Makris, C Hermans, ... Haemophilia 27 (1), 41-48, 2021 | 22* | 2021 |
Safety and efficacy of emicizumab and other novel agents in newborns and infants. GF Pierce, DP Hart, R Kaczmarek Haemophilia 25 (5), 2019 | 20* | 2019 |
Evaluation of an amino acid residue critical for the specificity and activity of human Gb3/CD77 synthase R Kaczmarek, K Mikolajewicz, K Szymczak, M Duk, E Majorczyk, ... Glycoconjugate Journal 33, 963-973, 2016 | 20 | 2016 |
Single nucleotide polymorphisms in A4GALT spur extra products of the human Gb3/CD77 synthase and underlie the P1PK blood group system R Kaczmarek, K Szymczak-Kulus, A Bereźnicka, K Mikołajczyk, M Duk, ... PLoS One 13 (4), e0196627, 2018 | 19 | 2018 |
Human Gb3/CD77 synthase produces P1 glycotope-capped N-glycans, which mediate Shiga toxin 1 but not Shiga toxin 2 cell entry K Szymczak-Kulus, S Weidler, A Bereznicka, K Mikolajczyk, R Kaczmarek, ... Journal of Biological Chemistry 296, 2021 | 18 | 2021 |