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Taking Care of the Caregivers: A Quality-of-Life Measure for the Parents of Children With Transfusion-Dependent 关爱护理者：一项针对输血依赖儿童父母的生存质量评估 Klaassen, Robert J.; Khelifi, Rania S.; Barrowman, Nicholas; Merelles-Pulcini, Manuela; Vichinsky, Elliott P.; Kirby-Allen, Melanie; Neufeld, Ellis J.; Kwiatkowski, Janet L.; Wu, John; Young, Nancy L. 分享 收藏
Trends in HU utilization and cerebrovascular outcomes before and after publication of the 2014 National Heart, Lung, and Blood Institute sickle cell disease guidelines Dain, Aleksandra S.; Li, Yimei; Master, Sahal; Bailey, Charles L.; Vossough, Arastoo; Ichord, Rebecca N.; Raffini, Leslie; Getz, Kelly; Kwiatkowski, Janet L. 分享 收藏
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An Update on Lovotibeglogene Autotemcel (Lovo-cel) Clinical Trials for Sickle Cell Disease (SCD) and Analysis of Early Predictors of Response to Lovo-Cel 关于Lovotibeglogene Autotemcel（Lovo-cel）治疗镰状细胞病（SCD）的临床试验进展及对Lovo-cel反应的早期预测因子分析 Rifkin-Zenenberg, Stacey; Kanter, Julie; Kinney, Melissa A.; Kwiatkowski, Janet L.; Nickel, Robert S.; Walters, Mark C.; Parikh, Suhag; Thompson, Alexis; George, Anil P.; Mapara, Markus; Martin, Paul L.; Chawla, Anjulika; Lodaya, Ankit; Pan, Lin; Sheldon-Waniga, Emily; Pierciey Jr, Francis J.; Tisdale, John; Gupta, Ashish O. 分享 收藏
Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial Betibeglogene autotemcel基因治疗输血依赖性，严重基因型 β-地中海贫血 (HGB-212) 患者: 一项非随机，多中心，单臂，开放标签，单剂量，3期试验 Kwiatkowski, Janet L.; Walters, Mark C.; Hongeng, Suradej; Yannaki, Evangelia; Kulozik, Andreas E.; Kunz, Joachim B.; Sauer, Martin G.; Thrasher, Adrian J.; Thuret, Isabelle; Lal, Ashutosh; Tao, Ge; Ali, Shamshad; Thakar, Himal L.; Elliot, Heidi; Lodaya, Ankit; Lee, Ji; Colvin, Richard A.; Locatelli, Franco; Thompson, Alexis A. 分享 收藏
Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia Locatelli, F.; Lang, P.; Wall, D.; Meisel, R.; Corbacioglu, S.; Li, A. M.; de la Fuente, J.; Shah, A. J.; Carpenter, B.; Kwiatkowski, J. L.; Mapara, M.; Liem, R. I.; Cappellini, M. D.; Algeri, M.; Kattamis, A.; Sheth, S.; Grupp, S.; Handgretinger, R.; Kohli, P.; Shi, D.; Ross, L.; Bobruff, Y.; Simard, C.; Zhang, L.; Morrow, P. K.; Hobbs, W. E.; Frangoul, H. 分享 收藏
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Deferiprone for transfusional iron overload in sickle cell disease and other anemias: open-label study of up to 3 years Elalfy, Mohsen S.; Hamdy, Mona; El-Beshlawy, Amal; Ebeid, Fatma S. E.; Badr, Mohamed; Kanter, Julie; Inusa, Baba; Adly, Amira A. M.; Williams, Suzan; Kilinc, Yurdanur; Lee, David; Fradette, Caroline; Rozova, Anna; Temin, Noemi Toiber; Tricta, Fernando; Kwiatkowski, Janet L. 分享 收藏
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Deferiprone vs deferoxamine for transfusional iron overload in SCD and other anemias: a randomized, open-label noninferiority study Kwiatkowski, Janet L.; Hamdy, Mona; El-Beshlawy, Amal; Ebeid, Fatma S. E.; Badr, Mohammed; Alshehri, Abdulrahman; Kanter, Julie; Inusa, Baba; Adly, Amira A. M.; Williams, Suzan; Kilinc, Yurdanur; Lee, David; Tricta, Fernando; Elalfy, Mohsen S. 分享 收藏
Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia Betibeglogene Autotemcel基因治疗非 β0/β0基因型 β 地中海贫血 Locatelli, Franco; Thompson, Alexis A.; Kwiatkowski, Janet L.; Porter, John B.; Thrasher, Adrian J.; Hongeng, Suradej; Sauer, Martin G.; Thuret, Isabelle; Lal, Ashutosh; Algeri, Mattia; Schneiderman, Jennifer; Olson, Timothy S.; Carpenter, Ben; Amrolia, Persis J.; Anurathapan, Usanarat; Schambach, Axel; Chabannon, Christian; Schmidt, Manfred; Labik, Ivan; Elliot, Heidi; Guo, Ruiting; Asmal, Mohammed; Colvin, Richard A.; Walters, Mark C. 分享 收藏
Inclusion of a short hairpin RNA targeting BCL11A into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin Lourenco, Silvia Pires; Jarocha, Danuta; Ghiaccio, Valentina; Guerra, Amaliris; Abdulmalik, Osheiza; La, Ping; Zezulin, Alexandra; Smith-Whitley, Kim; Kwiatkowski, Janet L.; Guzikowski, Virginia; Nakamura, Yukio; Raabe, Tobias; Breda, Laura; Rivella, Stefano 分享 收藏
Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies Breda, Laura; Ghiaccio, Valentina; Tanaka, Naoto; Jarocha, Danuta; Ikawa, Yasuhiro; Abdulmalik, Osheiza; Dong, Alisa; Casu, Carla; Raabe, Tobias D.; Shan, Xiaochuan; Danet-Desnoyers, Gwenn A.; Doto, Aoife M.; Everett, John; Bushman, Frederic D.; Radaelli, Enrico; Assenmacher, Charles A.; Tarrant, James C.; Hoepp, Natalie; Kurita, Ryo; Nakamura, Yukio; Guzikowski, Virginia; Smith-Whitley, Kim; Kwiatkowski, Janet L.; Rivella, Stefano 分享 收藏
Characterization of the severe phenotype of pyruvate kinase deficiency 丙酮酸激酶缺乏症严重表型的表征 Al-Samkari, Hanny; van Beers, Eduard J.; Morton, D. Holmes; Barcellini, Wilma; Eber, Stefan W.; Glader, Bertil; Yaish, Hassan M.; Chonat, Satheesh; Kuo, Kevin H. M.; Kollmar, Nina; Despotovic, Jenny M.; Pospisilova, Dagmar; Knoll, Christine M.; Kwiatkowski, Janet L.; Pastore, Yves D.; Thompson, Alexis A.; Wlodarski, Marcin W.; Ravindranath, Yaddanapudi; Rothman, Jennifer A.; Wang, Heng; Holzhauer, Susanne; Breakey, Vicky R.; Verhovsek, Madeleine M.; Kunz, Joachim; Sheth, Sujit; Sharma, Mukta; Rose, Melissa J.; Bradeen, Heather A.; McNaull, Melissa N.; Addonizio, Kathryn; Al-Sayegh, Hasan; London, Wendy B.; Grace, Rachael F. 分享 收藏
Safety and feasibility of hematopoietic progenitor stem cell collection by mobilization with plerixafor followed by apheresis vs bone marrow harvest in patients with sickle cell disease in the multi-center HGB-206 trial Tisdale, John F.; Pierciey, Francis J., Jr.; Bonner, Melissa; Thompson, Alexis A.; Krishnamurti, Lakshmanan; Mapara, Markus Y.; Kwiatkowski, Janet L.; Shestopalov, Ilya; Ribeil, Jean-Antoine; Huang, Wenmei; Asmal, Mohammed; Kanter, Julie; Walters, Mark C. 分享 收藏
Genotype-phenotype correlation and molecular heterogeneity in pyruvate kinase deficiency 丙酮酸激酶缺乏症的基因型-表型相关性和分子异质性 Bianchi, Paola; Fermo, Elisa; Lezon-Geyda, Kimberly; van Beers, Eduard J.; Morton, Holmes D.; Barcellini, Wilma; Glader, Bertil; Chonat, Satheesh; Ravindranath, Yaddanapudi; Newburger, Peter E.; Kollmar, Nina; Despotovic, Jenny M.; Verhovsek, Madeleine; Sharma, Mukta; Kwiatkowski, Janet L.; Kuo, Kevin H. M.; Wlodarski, Marcin W.; Yaish, Hassan M.; Holzhauer, Susanne; Wang, Heng; Kunz, Joachim; Addonizio, Kathryn; Al-Sayegh, Hasan; London, Wendy B.; Andres, Oliver; van Wijk, Richard; Gallagher, Patrick G.; Grace, Rachael F. F. 分享 收藏
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