Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura von Scully, Marie, Antun, Ana, Cataland, Spero R., Coppo, Paul, Dossier, Claire, Biebuyck, Nathalie, Hassenpflug, Wolf-Achim, Kentouche, Karim, Knöbl, Paul, Kremer Hovinga, Johanna A., López-Fernández, M. Fernanda, Matsumoto, Masanori, Ortel, Thomas L., Windyga, Jerzy, Bhattacharya, Indranil, Cronin, Michael, Li, Hong, Mellgård, Björn, Patel, Munjal, Patwari, Parth, Xiao, Shan, Zhang, Pinghai, Wang, Linda T.

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Reductions in glucosylsphingosine (lyso-Gb1) in treatment-naïve and previously treated patients receiving velaglucerase alfa for type 1 Gaucher disease: Data from phase 3 clinical... von Elstein, Deborah, Mellgard, Björn, Dinh, Quinn, Lan, Lan, Qiu, Yongchang, Cozma, Claudia, Eichler, Sabrina, Böttcher, Tobias, Zimran, Ari

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Recombinant ADAMTS13 for the Treatment of Acute TTP Events in Patients with Congenital Thrombotic Thrombocytopenic Purpura: Results from the Phase 3 Randomized, Controlled, Crossov... von Scully, Marie, Ortel, Thomas L., Yu, Ziqiang, Waliullah, Maria, Zhang, Pinghai, Patel, Munjal, Patwari, Parth, Mellgard, Bjorn, Wang, Linda T.

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Pregnancy-Related Outcomes in Patients with Congenital Thrombotic Thrombocytopenic Purpura: Post Hoc Analysis of a Retrospective Chart Review Study von Coppo, PAUL, Scully, Marie, Kremer Hovinga, Johanna A., Jose Aragon, Maria, Patwari, Parth, Wang, Linda T., Mellgard, Bjorn, Ayodele, Olulade, Saad, Ragy

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Clinical Outcomes of Congenital Thrombotic Thrombocytopenic Purpura: A Multinational Chart Review Study von Coppo, PAUL, Scully, Marie, Kremer Hovinga, Johanna A., Jose Aragon, Maria, Patwari, Parth, Wang, Linda T., Mellgard, Bjorn, Ayodele, Olulade, Saad, Ragy

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A multicenter, open-label extension study of velaglucerase alfa in Japanese patients with Gaucher disease: Results after a cumulative treatment period of 24 months von Ida, Hiroyuki, Tanaka, Akemi, Matsubayashi, Tomoko, Murayama, Kei, Hongo, Teruaki, Lee, Hak-Myung, Mellgard, Björn

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Prevalence of Vaso-Occlusive Crises in Patients with Sickle Cell Disease: A Retrospective US Claims Database Analysis von Oladapo, Abiola, Swallow, Elyse, Briggs, Allison, Zichlin, Miriam L., Mellgard, Bjorn L

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Recombinant ADAMTS13 reduces abnormally up-regulated von Willebrand factor in plasma from patients with severe COVID-19 von Turecek, Peter L., Peck, Rachel C., Rangarajan, Savita, Reilly-Stitt, Christopher, Laffan, Michael A., Kazmi, Rashid, James, Izabela, Dushianthan, Ahilanandan, Schrenk, Gerald, Gritsch, Herbert, Ewenstein, Bruce M., Mellgard, Bjorn, Erdlenbruch, Wolfhard, Jain, Nisha, Binder, Nikolaus B., Mumford, Andrew D.

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Home infusion of intravenous velaglucerase alfa: Experience from pooled clinical studies in 104 patients with type 1 Gaucher disease von Elstein, Deborah, Burrow, T Andrew, Charrow, Joel, Giraldo, Pilar, Mehta, Atul, Pastores, Gregory M, Lee, Hak-Myung, Mellgard, Björn, Zimran, Ari

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Recombinant von Willebrand factor prophylaxis in patients with severe von Willebrand disease: phase 3 study results von Leebeek, Frank W G, Peyvandi, Flora, Escobar, Miguel, Tiede, Andreas, Castaman, Giancarlo, Wang, Michael, Wynn, Tung, Baptista, Jovanna, Wang, Yi, Zhang, Jingmei, Mellgård, Björn, Özen, Gülden

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Genetic variation in DLG5 is associated with inflammatory bowel disease von Lagerstrom-Fermer, Maria, Hampe, Jochen, Rosenstiel, Philip, Stoll, Monika, Albrecht, Mario, Corneliussen, Brit, Foelsch, Ulrich R, Schreiber, Stefan, Croucher, Peter J P, Waetzig, Georg H, Lengauer, Thomas, Costello, Christine M, Mellgard, Bjorn, Seegert, Dirk, Mathew, Christopher G, Nikolaus, Susanna, Koch, W Andreas, Pierrou, Stefan

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Results From a 12-Month Open-Label Phase 1/2 Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease von Tantawy, Azza A. G., El-Beshlawy, Amal, Marzouk, Iman, Bavdekar, Ashish, Qin, Yulin, Mellgard, Björn, Ben Turkia, Hadhami

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Structure and Function of Recombinant versus Plasma-Derived von Willebrand Factor and Impact on Multimer Pharmacokinetics in von Willebrand Disease von Gritsch, Herbert, Schrenk, Gerald, Weinhappl, Nina, Mellgard, Bjorn, Ewenstein, Bruce, Turecek, Peter L

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Effects of velaglucerase alfa on bone-related pathology in patients with type 1 Gaucher disease: Design and methods of a 2-year open-label phase 4 study von Mellgard, Björn, Hughes, Derralynn, Qin, Yulin, Deegan, Patrick

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A comparison of oligonucleotide and cDNA-based microarray systems von Mah, Nancy, Thelin, Anders, Lu, Tim, Nikolaus, Susanna, Kuhbacher, Tanja, Gurbuz, Yesim, Eickhoff, Holger, Kloppel, Gunther, Lehrach, Hans, Mellgard, Bjorn, Costello, Christine M, Schreiber, Stefan

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P1602: ACUTE EPISODES IN PATIENTS WITH CONGENITAL THROMBOTIC THROMBOCYTOPENIC PURPURA RECEIVING PROPHYLAXIS: POST HOC ANALYSIS OF A RETROSPECTIVE CHART REVIEW STUDY von Coppo, Paul, Scully, Marie, Hovinga, Johanna Kremer, Tarasco, Erika, Willock, Rosa, Aragon, Maria Jose, Patwari, Parth, Wang, Linda T., Cronin, Michael, Mellgård, Björn, Ayodele, Olulade, Du, Ping

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METHODS OF PROPHYLACTIC TREATMENT USING RECOMBINANT VWF (RVWF) von EWENSTEIN, BRUCE, MELLGARD, BJORN

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A multicenter, open-label extension study of velaglucerase alfa in Japanese patients with Gaucher disease: Results after a cumulative treatment period of 24months von Ida, Hiroyuki, Tanaka, Akemi, Matsubayashi, Tomoko, Murayama, Kei, Hongo, Teruaki, Lee, Hak-Myung, Mellgard, Björn

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S305: PHASE 2 RANDOMIZED, PLACEBO‐CONTROLLED, DOUBLE‐BLIND, MULTICENTER STUDY OF RECOMBINANT ADAMTS13 IN PATIENTS WITH IMMUNE‐MEDIATED THROMBOTIC THROMBOCYTOPENIC PURPURA von Scully, Marie, Baptista, Jovanna, Bhattacharya, Indranil, Cataland, Spero, Coppo, Paul, Cuccia, Loredana, Dutt, Tina, Susan Huang, Shih‐Han, Pascual Izquierdo, Cristina, Eva Mingot‐Castellano, María, Parnes, Aric, Pavenski, Katerina, Rajavel, Kavitha, Jarque, Isidro, Wang, Linda T., Fernández Zarzoso, Miguel, Zhu, Andy, Mellgård, Björn

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Velaglucerase alfa enzyme replacement therapy in children and adolescents with type 3 Gaucher disease: Results of a 12-month multicenter, open-label phase 1/2 study von Tantawy, Azza A.G., El-Beshlawy, Amal, Marzouk, Iman, Bavdekar, Ashish, Qin, Yulin, Mellgard, Björn, Turkia, Hadhami Ben

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