NGLY1 deficiency: estimated incidence, clinical features, and genotypic spectrum from the NGLY1 Registry von Stanclift, Caroline R, Dwight, Selina S, Lee, Kevin, Eijkenboom, Quirine L, Wilsey, Matt, Wilsey, Kristen, Kobayashi, Erica Sanford, Tong, Sandra, Bainbridge, Matthew N

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Novel p38α Mitogen-Activated Protein Kinase Inhibitor Shows Analgesic Efficacy in Acute Postsurgical Dental Pain von Tong, Sandra E., Daniels, Stephen E., Black, Peter, Chang, Stephen, Protter, Andrew, Desjardins, Paul J.

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The measurement of physical functioning among patients with Tenosynovial Giant Cell Tumor (TGCT) using the Patient-Reported Outcomes Measurement Information System (PROMIS) von Gelhorn, Heather L., Ye, Xin, Speck, Rebecca M., Tong, Sandra, Healey, John H., Bukata, Susan V., Lackman, Richard D., Murray, Lindsey, Maclaine, Grant, Lenderking, William R., Hsu, Henry H., Lin, Paul S., Tap, William D.

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Randomized Trial on the Effect of an Oral Spleen Tyrosine Kinase Inhibitor in the Treatment of IgA Nephropathy von Tam, Frederick W.K., Tumlin, James, Barratt, Jonathan, Rovin, Brad H., Roberts, Ian S.D., Roufosse, Candice, Cook, H. Terence, Bhangal, Gurjeet, Brown, Alison L., Busch, Martin, Dudhiya, Fayaz, Duliege, Anne-Marie, Fraser, Donald J., Gale, Daniel P., Huang, Chiu-Ching, Lai, Ping-Chin, Lee, Meng, Masuda, Esteban S., McAdoo, Stephen P., Rosenkranz, Alexander R., Sommerer, Claudia, Sunder-Plassmann, Gere, Szeto, Cheuk-Chun, Tang, Sydney C.W., Williamson, Don E., Willcocks, Lisa, Vielhauer, Volker, Kim, Min Jeong, Todd, Leslie, Zayed, Hany, Tong-Starksen, Sandra, Lafayette, Richard

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Clinical Trial to Evaluate an Approved ITP Therapy Targeting Spleen Tyrosine Kinase (SYK) for Prevention and Treatment of COVID-19 Related Complications von Dummer, Wolfgang, Markovtsov, Vadim V., Tong, Sandra, Masuda, Esteban

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Long-Term Safety Profile of the Oral Spleen Tyrosine Kinase Inhibitor Fostamatinib in Immune Thrombocytopenia (ITP) and Other Diseases von Tong, Sandra, Numerof, Robert P., Datangel, Jane, Masuda, Esteban

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Fostamatinib for the Treatment of Warm Antibody Autoimmune Hemolytic Anemia (wAIHA): A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study von Cooper, Nichola, Numerof, Robert P., Tong, Sandra, Kuter, David J.

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Structure-Guided Blockade of CSF1R Kinase in Tenosynovial Giant-Cell Tumor von Tap, William D, Wainberg, Zev A, Anthony, Stephen P, Ibrahim, Prabha N, Zhang, Chao, Healey, John H, Chmielowski, Bartosz, Staddon, Arthur P, Cohn, Allen Lee, Shapiro, Geoffrey I, Keedy, Vicki L, Singh, Arun S, Puzanov, Igor, Kwak, Eunice L, Wagner, Andrew J, Von Hoff, Daniel D, Weiss, Glen J, Ramanathan, Ramesh K, Zhang, Jiazhong, Habets, Gaston, Zhang, Ying, Burton, Elizabeth A, Visor, Gary, Sanftner, Laura, Severson, Paul, Nguyen, Hoa, Kim, Marie J, Marimuthu, Adhirai, Tsang, Garson, Shellooe, Rafe, Gee, Carolyn, West, Brian L, Hirth, Peter, Nolop, Keith, van de Rijn, Matt, Hsu, Henry H, Peterfy, Charles, Lin, Paul S, Tong-Starksen, Sandra, Bollag, Gideon

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Long‐term fostamatinib treatment of adults with immune thrombocytopenia during the phase 3 clinical trial program von Bussel, James B., Arnold, Donald M., Boxer, Michael A., Cooper, Nichola, Mayer, Jiri, Zayed, Hany, Tong, Sandra, Duliege, Anne‐Marie

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Fostamatinib is an effective second‐line therapy in patients with immune thrombocytopenia von Boccia, Ralph, Cooper, Nichola, Ghanima, Waleed, Boxer, Michael A, Hill, Quentin A., Sholzberg, Michelle, Tarantino, Michael D., Todd, Leslie K., Tong, Sandra, Bussel, James B.

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Fostamatinib for the treatment of diffuse large B-cell lymphoma von Arkenau, Hendrik-Tobias, Patrikidou, Anna, Flinn, Ian, Hylton, Jonas C., Tong, Sandra, Ardeshna, Kirit

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561. Phase 3 Trial of Fostamatinib for the Treatment of COVID-19: Repurposing an Immunomodulatory Drug Previously Approved for Immune Thrombocytopenia von Mallat, Ziad, Lobo, Suzana Margareth, Malik, Anuj, Tong, Sandra

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Fostamatinib for the treatment of warm antibody autoimmune hemolytic anemia: Phase 2, multicenter, open‐label study von Kuter, David J., Rogers, Kerry A., Boxer, Michael A., Choi, Michael, Agajanian, Richy, Arnold, Donald M., Broome, Catherine M., Field, Joshua J., Murakhovskaya, Irina, Numerof, Robert, Tong, Sandra

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NGLY1 deficiency: a prospective natural history study von Tong, Sandra, Ventola, Pamela, Frater, Christina H, Klotz, Jenna, Phillips, Jennifer M, Muppidi, Srikanth, Dwight, Selina S, Mueller, William F, Beahm, Brendan J, Wilsey, Matt, Lee, Kevin J

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Patient-reported Symptoms of Tenosynovial Giant Cell Tumors von Gelhorn, Heather L., PhD, Tong, Sandra, MD, McQuarrie, Kelly, BS, Vernon, Christina, MPH, Hanlon, Jennifer, MPH, Maclaine, Grant, BMedSc, MBBS, MEc, MA, Lenderking, William, PhD, Ye, Xin, PhD, MS, Speck, Rebecca M., PhD, MPH, Lackman, Richard D., MD, Bukata, Susan V., MD, Healey, John H., MD, Keedy, Vicki L., MD, MSCI, Anthony, Stephen P., DO, Wagner, Andrew J., MD, PhD, Von Hoff, Daniel D., MD, Singh, Arun S., MD, Becerra, Carlos R., MD, Hsu, Henry H., MD, Lin, Paul S., MD, MBA, Tap, William D., MD

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Two-Year Safety and Efficacy Outcomes with Fostamatinib in Adult Patients with Immune Thrombocytopenia (ITP): Open-Label Extension to Phase 3 Trial Program von Duliege, Anne-Marie, Arnold, Donald M., Boccia, Ralph, Boxer, Michael, Cooper, Nichola, Hill, Quentin A, Liles, Darla K, Sholzberg, Michelle, Zayed, Hany, Tong, Sandra, Bussel, James B.

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Abstract B176: Results of a phase I study of PLX8394, a next-generation BRAF inhibitor, in refractory solid tumors von Janku, Filip, Vaishampayan, Ulka, Khemka, Vivek, Bhatty, Minny, Zhang, Chao, Hsu, Henry H., Lin, Paul S., Tong, Sandra, Sharma, Sunil

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Two placebo-controlled phase 3 studies of fostamatinib, an oral spleen tyrosine kinase (Syk) inhibitor, for the treatment of persistent/chronic immune thrombocytopenia (ITP) in adu... von Bussel, James B., Arnold, Donald, Cooper, Nichola, Abdallah, Alhossain, Boxer, Michael, Liles, Darla K., Ghanima, Waleed, McCrae, Keith, Zayed, Hany, Tong, Sandra, Cadieux, Ben, Duliege, Anne-Marie

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Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial von Tap, William D, Gelderblom, Hans, Palmerini, Emanuela, Desai, Jayesh, Bauer, Sebastian, Blay, Jean-Yves, Alcindor, Thierry, Ganjoo, Kristen, Martín-Broto, Javier, Ryan, Christopher W, Thomas, David M, Peterfy, Charles, Healey, John H, van de Sande, Michiel, Gelhorn, Heather L, Shuster, Dale E, Wang, Qiang, Yver, Antoine, Hsu, Henry H, Lin, Paul S, Tong-Starksen, Sandra, Stacchiotti, Silvia, Wagner, Andrew J

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Fostamatinib, a Spleen Tyrosine Kinase Inhibitor, for the Treatment of Warm Antibody Autoimmune Hemolytic Anemia: Initial Results of the Multicenter, Open-Label Extension Period of... von Kuter, David J., Agajanian, Richy, Arnold, Donald M., Boxer, Michael, Broome, Catherine, Field, Joshua J., Murakhovskaya, Irina, Rogers, Kerry A., Lowe, Ann M, Tong, Sandra, Zayed, Hany, Duliege, Anne-Marie

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