Personal profile
Research interests
My work is focused on understanding the etiologies of thrombotic microangiopathies to effect a treatment. We have studied the structure function relationship of von Willebrand Factor and ADAMTS13. Deficiency of ADAMTS13 activity leads to a thrombotic microangiopathy. We have identified a small polypeptide of ADAMTS13 that will enhance cleavage of von Willebrand Factor by full-length ADAMTS13. We have also participated in the clinical trials of eculizumab in the treatment of atypical hemolytic uremic syndrome, another thrombotic microangiopathy that results from deficient complement regulation.
Clinical interests
Blood disorders, hemophilia and bleeding disorders, disorders of blood-forming tissues, hemostasis, thrombosis, hematology, cardiovascular metabolic research; platelets and coagulation factors
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Collaborations and top research areas from the last five years
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Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial results
Leavitt, A. D., Mahlangu, J., Raheja, P., Symington, E., Quon, D. V., Giermasz, A., Kenet, G., Lowe, G., Key, N. S., Millar, C. M., Pipe, S. W., Chou, S. C., Klamroth, R., Mason, J., Chambost, H., Peyvandi, F., Majerus, E., Pepperell, D., Tan, C. W. & Yu, H. & 2 others, , Mar 2026, In: Research and Practice in Thrombosis and Haemostasis. 10, 3, 103416.Research output: Contribution to journal › Article › peer-review
Open Access1 Link opens in a new tab Scopus citations -
Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial
Leavitt, A. D., Mahlangu, J., Raheja, P., Symington, E., Quon, D. V., Giermasz, A., López Fernández, M. F., Kenet, G., Lowe, G., Key, N. S., Millar, C. M., Pipe, S. W., Madan, B., Chou, S. C., Klamroth, R., Mason, J., Chambost, H., Peyvandi, F., Majerus, E. & Pepperell, D. & 4 others, , Nov 2024, In: Research and Practice in Thrombosis and Haemostasis. 8, 8, 102615.Research output: Contribution to journal › Article › peer-review
Open Access36 Link opens in a new tab Scopus citations -
Pegcetacoplan in paroxysmal nocturnal haemoglobinuria: Its use, its clinical effectiveness, and its influence on health-related quality of life and productivity
Wilson, K., Rich, C., Hakimi, Z., Horneff, R., Fishman, J., Mellor, J., Earl, L., Taylor, Y., Simons, A., Conyers, J., Mulherin, B., Majerus, E. & Röth, A., Apr 2024, In: European Journal of Haematology. 112, 4, p. 516-529 14 p.Research output: Contribution to journal › Article › peer-review
Open Access7 Link opens in a new tab Scopus citations -
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, A. L., Leavitt, A. D., Peyvandi, F., Ozelo, M. C., Mahlangu, J., Peerlinck, K., Wang, J. D., Lowe, G. C., Tan, C. W., Giermasz, A., Tran, H., Khoo, T. L., Cockrell, E., Pepperell, D., Chambost, H., López Fernández, M. F., Kazmi, R., Majerus, E. & Skinner, M. W. & 6 others, , Dec 2023, In: Journal of Thrombosis and Haemostasis. 21, 12, p. 3450-3462 13 p.Research output: Contribution to journal › Article › peer-review
Open Access23 Link opens in a new tab Scopus citations -
Valoctocogene roxaparvovec gene transfer in participants with HIV
Ragni, M. V., Majerus, E., Fong, S., Yates, B., Scheeler, S., Razon, L., Yu, H., Reddy, D. B. & Robinson, T. M., Apr 25 2023, In: Blood Advances. 7, 8, p. 1525-1530 6 p.Research output: Contribution to journal › Letter › peer-review
Open Access12 Link opens in a new tab Scopus citations