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Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency Migliavacca, Maddalena; Barzaghi, Federica; Fossati, Claudia; Rancoita, Paola M. V.; Gabaldo, Michela; Dionisio, Francesca; Giannelli, Stefania; Salerio, Federica Andrea; Ferrua, Francesca; Tucci, Francesca; Calbi, Valeria; Gallo, Vera; Recupero, Salvatore; Consiglieri, Giulia; Pajno, Roberta; Sambuco, Maria; Priolo, Alessio; Ferri, Chiara; Garella, Vittoria; Monti, Ilaria; Silvani, Paolo; Darin, Silvia; Casiraghi, Miriam; Corti, Ambra; Zancan, Stefano; Levi, Margherita; Cesana, Daniela; Carlucci, Filippo; Pituch-Noworolska, Anna; Abdelaziz, Dalia; Baumann, Ulrich; Finocchi, Andrea; Cancrini, Caterina; Ladogana, Saverio; Meinhardt, Andrea; Meyts, Isabelle; Montin, Davide; Notarangelo, Lucia Dora; Porta, Fulvio; Pasquet, Marlene; Speckmann, Carsten; Stepensky, Polina; Tommasini, Alberto; Rabusin, Marco; Karakas, Zeynep; Galicchio, Miguel; Leonardi, Lucia; Duse, Marzia; Guner, Sukru Nail; Di Serio, Clelia; Ciceri, Fabio; Bernardo, Maria Ester; Aiuti, Alessandro; Cicalese, Maria Pia 分享 收藏
The long-term efficacy and safety of nilotinib in pediatric patients with CML: a 5-year update of the DIALOG study Hijiya, Nobuko; Maschan, Alexey; Rizzari, Carmelo; Shimada, Hiroyuki; Dufour, Carlo; Goto, Hiroaki; Kang, Hyoung Jin; Guinipero, Terri; Karakas, Zeynep; Bautista, Francisco; Ducassou, Stephane; Yoo, Keon Hee; Zwaan, Christian Michel; Millot, Frederic; Patterson, Briana C.; Samis, Jill; Izquierdo, Miguel; Titorenko, Ksenia; Li, Sai; Sosothikul, Darintr 分享 收藏
An open-label, multicenter, efficacy, and safety study of deferasirox in iron-overloaded patients with non-transfusion-dependent thalassemia (THETIS): 5-year results Lai, Yong-Rong; Cappellini, Maria Domenica; Aydinok, Yesim; Porter, John; Karakas, Zeynep; Viprakasit, Vip; Siritanaratkul, Noppadol; Kattamis, Antonis; Liu, Rongrong; Izquierdo, Miguel; Lasher, Janet; Govindaraju, Sameera; Taher, Ali 分享 收藏
A phase 2 study of nilotinib in pediatric patients with CML: long-term update on growth retardation and safety Hijiya, Nobuko; Maschan, Alexey; Rizzari, Carmelo; Shimada, Hiroyuki; Dufour, Carlo; Goto, Hiroaki; Kang, Hyoung Jin; Guinipero, Terri; Karakas, Zeynep; Bautista, Francisco; Ducassou, Stephane; Yoo, Keon Hee; Zwaan, Christian Michel; Millot, Frederic; Patterson, Briana; Samis, Jill; Aimone, Paola; Allepuz, Alex; Titorenko, Ksenia; Sosothikul, Darintr 分享 收藏
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Effect of Poloxamer 188 vs Placebo on Painful Vaso-Occlusive Episodes in Children and Adults With Sickle Cell Disease: A Randomized Clinical Trial Casella, James F.; Barton, Bruce A.; Kanter, Julie; Black, L. Vandy; Majumdar, Suvankar; Inati, Adlette; Wali, Yasser; Drachtman, Richard A.; Abboud, Miguel R.; Kilinc, Yurdanur; Fuh, Beng R.; Al-Khabori, Murtadha K.; Takemoto, Clifford M.; Salman, Emad; Sarnaik, Sharada A.; Shah, Nirmish; Morris, Claudia R.; Keates-Baleeiro, Jennifer; Raj, Ashok; Alvarez, Ofelia A.; Hsu, Lewis L.; Thompson, Alexis A.; Sisler, India Y.; Pace, Betty S.; Noronha, Suzie A.; Lasky, Joseph L., III; de Julian, Elena Cela; Godder, Kamar; Thornburg, Courtney Dawn; Kamberos, Natalie L.; Nuss, Rachelle; Marsh, Anne M.; Owen, William C.; Schaefer, Anne; Tebbi, Cameron K.; Chantrain, Christophe F.; Cohen, Debra E.; Karakas, Zeynep; Piccone, Connie M.; George, Alex; Fixler, Jason M.; Singleton, Tammuella C.; Moulton, Thomas; Quinn, Charles T.; de Castro Lobo, Clarisse Lopes; Almomen, Abdulkareem M.; Goyal-Khemka, Meenakshi; Maes, Philip; Emanuele, Marty; Gorney, Rebecca T.; Padgett, Claire S.; Parsley, Ed; Kronsberg, Shari S.; Kato, Gregory J.; Gladwin, Mark T. 分享 收藏
Safety and efficacy of anticoagulant therapy in pediatric catheter-related venous thrombosis (EINSTEIN-Jr CVC-VTE) Thom, Katharina; Lensing, Anthonie W. A.; Nurmeev, Ildar; Bajolle, Fanny; Bonnet, Damien; Kenet, Gili; Massicotte, M. Patricia; Karakas, Zeynep; Palumbo, Joseph S.; Saracco, Paola; Amedro, Pascal; Chain, Juan; Chan, Anthony K.; Ikeyama, Takanari; Lam, Joyce C. M.; Gauger, Cynthia; Pap, Akos Ferenc; Majumder, Madhurima; Kubitza, Dagmar; Smith, William T.; Berkowitz, Scott D.; Prins, Martin H.; Monagle, Paul; Young, Guy; Male, Christoph 分享 收藏
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Successful haematopoietic stem cell transplantation in 44 children from healthy siblings conceived after preimplantation HLA matching Kahraman, Semra; Beyazyurek, Cagri; Yesilipek, Mehmet Akif; Ozturk, Gulyuz; Ertem, Mehmet; Anak, Sema; Kansoy, Savas; Aksoylar, Serap; Kuskonmaz, Baris; Oniz, Haldun; Slavin, Shimon; Karakas, Zeynep; Tac, Huseyin Avni; Gulum, Nese; Ekmekci, Gokhan Cumhur 分享 收藏
Deregulated WNT signaling in childhood T-cell acute lymphoblastic leukemia Ng, O. H.; Erbilgin, Y.; Firtina, S.; Celkan, T.; Karakas, Z.; Aydogan, G.; Turkkan, E.; Yildirmak, Y.; Timur, C.; Zengin, E.; van Dongen, J. J. M.; Staal, F. J. T.; Ozbek, U.; Sayitoglu, M. 分享 收藏
New compliant strain gauges for self-sensing dynamic deformation of flapping wings on miniature air vehicles Wissman, James; Perez-Rosado, Ariel; Edgerton, Alex; Levi, Benjamin M.; Karakas, Zeynep N.; Kujawski, Mark; Philipps, Alyssa; Papavizas, Nicholas; Fallon, Danielle; Bruck, Hugh A.; Smela, Elisabeth 分享 收藏
Deferasirox effectively reduces iron overload in non-transfusion-dependent thalassemia (NTDT) patients: 1-year extension results from the THALASSA study Taher, Ali T.; Porter, John B.; Viprakasit, Vip; Kattamis, Antonis; Chuncharunee, Suporn; Sutcharitchan, Pranee; Siritanaratkul, Noppadol; Galanello, Renzo; Karakas, Zeynep; Lawniczek, Tomasz; Habr, Dany; Ros, Jacqueline; Zhu, Zewen; Cappellini, M. Domenica 分享 收藏
Deferasirox demonstrates a dose-dependent reduction in liver iron concentration and consistent efficacy across subgroups of non-transfusion-dependent thalassemia patients Taher, Ali T.; Porter, John B.; Viprakasit, Vip; Kattamis, Antonis; Chuncharunee, Suporn; Sutcharitchan, Pranee; Siritanaratkul, Noppadol; Galanello, Renzo; Karakas, Zeynep; Lawniczek, Tomasz; Habr, Dany; Ros, Jacqueline; Zhang, Yiyun; Cappellini, M. Domenica 分享 收藏
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