Bosutinib versus Placebo for Autosomal Dominant Polycystic Kidney Disease von Tesar, Vladimir, Ciechanowski, Kazimierz, Pei, York, Barash, Irina, Shannon, Megan, Li, Ray, Williams, Jason H, Levisetti, Matteo, Arkin, Steven, Serra, Andreas

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Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Domagrozumab (PF‐06252616), an Antimyostatin Monoclonal Antibody, in Healthy Subjects von Bhattacharya, Indranil, Pawlak, Sylvester, Marraffino, Shannon, Christensen, Jared, Sherlock, Sarah P., Alvey, Christine, Morris, Carl, Arkin, Steven, Binks, Michael

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Priority Outcomes in Sickle Cell Disease Treatment: Co-Creation and Implementation of a Preference Exercise With Patients and Caregivers to Inform Drug Development von Jalowsky, Maggie, Hauber, Brett, Scott, Mariah Jacqueline, Arkin, Steven, Coulter, Joshua R., Watt, Stephen J, Kelly, L Mariah G, Valentine, Ashley

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Practical and Statistical Considerations for the Long Term Follow‐Up of Gene Therapy Trial Participants von Rohde, Maximilian, Huh, Seoan, D'Souza, Vanessa, Arkin, Steven, Roberts, Erika, McIntosh, Avery

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Peak Thrombin and D-Dimer Levels in Subjects with Severe Hemophilia Receiving Acute Treatment for Bleeding Episodes Experienced during Prophylactic Marstacimab Treatment von Nayak, Satyaprakash, Raje, Sangeeta, Teeter, John, Harnisch, Lutz, Arkin, Steven

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Acute Events Significantly Increase Risk for Long-Term End-Organ Sequelae in Sickle Cell Disease - a Retrospective Cohort Study Using Optum Electronic Health Records von Sun, Xunming, Arkin, Steven, Gu, Yun, Schachterle, Stephen E, Zhou, Xiaofeng, Edwards, Thomas, Xiao, Sherry, Huang, Kui A

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Long‐term safety and efficacy of the anti‐tissue factor pathway inhibitor marstacimab in participants with severe haemophilia: Phase II study results von Mahlangu, Johnny, Luis Lamas, Jose, Cristobal Morales, Juan, Malan, Daniel R., Teeter, John, Charnigo, Robert J., Hwang, Eunhee, Arkin, Steven

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Evolution of Antidrug Antibody Assays During the Development of Anti-Tissue Factor Pathway Inhibitor Monoclonal Antibody Marstacimab von Donley, Jean, Jani, Darshana, Zhu, Tong, Xiang, Yuhong, Gorovits, Boris, Arkin, Steven

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Safety and efficacy of bosutinib (SKI-606) in chronic phase Philadelphia chromosome–positive chronic myeloid leukemia patients with resistance or intolerance to imatinib von Cortes, Jorge E., Kantarjian, Hagop M., Brümmendorf, Tim H., Kim, Dong-Wook, Turkina, Anna G., Shen, Zhi-Xiang, Pasquini, Ricardo, Khoury, H. Jean, Arkin, Steven, Volkert, Angela, Besson, Nadine, Abbas, Richat, Wang, Junyuan, Leip, Eric, Gambacorti-Passerini, Carlo

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Progress: Responsiveness Validation of an Electronic Patient Reported Outcome (ePRO) in Adults with Sickle Cell Disease (NCT05407805) von Minniti, Caterina P, Bronte-Hall, Lanetta, Marraffino, Shannon, Healy, Jason, Rybin, Denis, Beidler, David, Huang, Lori, Shelton, Dana, Taylor, Steven, Goossens, Colet, Arkin, Steven

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Effects of stathmin inhibition on the mitotic spindle von Iancu, C, Mistry, S J, Arkin, S, Wallenstein, S, Atweh, G F

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A phase 1b/2 clinical study of marstacimab, targeting human tissue factor pathway inhibitor, in haemophilia von Mahlangu, Johnny N., Lamas, Jose Luis, Morales, Juan Cristobal, Malan, Daniel R., Šalek, Silva Zupančić, Wang, Michael, Boggio, Lisa N., Hegemann, Inga, Mital, Andrzej, Cardinal, Matthew, Zhu, Tong, Sun, Pengling, Arkin, Steven

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The Effect of Tucaresol on Translational MRI Estimates of Cerebral Physiology in Townes SCD Mice von Goodman, James A., Yen, Yi-Fen, Chen, Yinching Iris, Chan, Suk-Tak, Weigand Wittier, Jonah P., Janz, Jay M., Arkin, Steven, Rybin, Denis, Knee, Kelly Marie, Pittman, Debra D, Mandeville, Joseph B.

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Four-Year Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia Α von Harrington, Thomas J., Giermasz, Adam, Visweshwar, Nathan, Leavitt, Andrew D., Konkle, Barbara A., Rupon, Jeremy, Dirusso, Gregory, Tseng, Li-Jung, de los Angeles Resa, Maria, Ganne, Florence, Agathon, Delphine, Plonski, Frank, Rouy, Didier, Cockroft, Bettina M., Fang, Annie F., Arkin, Steven

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Validation of Patient-Reported Vaso-Occlusive Crisis Day As a Potential Endpoint in Studies of Sickle Cell Disease von Coyne, Karin, Currie, Brooke, Callaghan, Michael U., Wyrwich, Kathleen W., Pease, Sheryl, Baker, Christine L., Arkin, Steven, Pittman, Debra D.

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Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia Α von Giermasz, Adam, Visweshwar, Nathan, Harrington, Thomas J., Leavitt, Andrew D, Konkle, Barbara, Rupon, Jeremy, Di Russo, Gregory, Tseng, Li-Jung, de los Angeles Resa, Maria, Ganne, Florence, Agathon, Delphine, Plonski, Frank, Rouy, Didier, Cockroft, Bettina M., Fang, Annie, Arkin, Steven

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Mechanical Thrombectomy for Acute Ischemic Stroke : Final Results of the Multi MERCI Trial von SMITH, Wade S, SUNG, Gene, GOBIN, Y. Pierre, SAVER, Jeffrey, BUDZIK, Ronald, DUCKWILER, Gary, LIEBESKIND, David S, LUTSEP, Helmi L, RYMER, Marilyn M, HIGASHIDA, Randall T, STARKMAN, Sidney

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The quintessence of immunogenicity reporting for biotherapeutics von Shankar, Gopi, Arkin, Steven, Devanarayan, Viswanath, Kromminga, Arno, Quarmby, Valerie, Richards, Susan, Subramanyam, Meena, Swanson, Steven

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Phase 1b Study to Evaluate Safety, Tolerability, and Maximum Tolerated Dose of PF-05230907 for Intracerebral Hemorrhage von Silva Blas, Yolanda, Diringer, Michael N., Lo, Benjamin, Masjuan, Jaime, Pérez de la Ossa, Natalia, Cardinal, Matthew, Yong, Florence, Zhu, Tong, Li, Gang, Arkin, Steven

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Updated Follow-up of the Alta Study, a Phase 1/2, Open Label, Adaptive, Dose-Ranging Study to Assess the Safety and Tolerability of SB-525 Gene Therapy in Adult Patients with Sever... von Konkle, Barbara A., Stine, Kimo, Visweshwar, Nathan, Harrington, Thomas J., Leavitt, Andrew D., Giermasz, Adam, Arkin, Steven, Di Russo, Gregory, Snyder, Ashley, Woolfson, Adrian, Rouy, Didier

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