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Efficacy and Safety of Subcutaneous Efgartigimod PH20 in Adults With Primary Immune Thrombocytopenia (ADVANCE SC): A Multicenter, Randomized, Double‐Blinded, Placebo‐Controlled, Phase 3 Trial
American Journal of Hematology, EarlyView.ABSTRACT
Primary autoimmune thrombocytopenia (ITP) is characterized by thrombocytopenia, bleeding, and reduced health‐related quality of life. In the Phase 3 ADVANCE IV study, intravenous efgartigimod induced significant platelet count responses versus placebo in patients with chronic ITP. ADVANCE SC, a Phase 3, multicenter, randomized, double‐blinded, Nichola Cooper, Catherine M. Broome, Yoshitaka Miyakawa, Vickie McDonald, Hanny Al‐Samkari, Abderrahim Khelif, Spero R. Cataland, Wilma Barcellini, Renchi Yang, Heng Mei, Filip Matthijssens, Anna Hultberg, Giorgia Ciurlia, Domenica Gandini, Jaume Ayguasanosa, Kristof De Beuf, Ségolène Pastouret, Waleed Ghanima, Francesco Rodeghiero, James B. Bussel, the ADVANCE SC Investigator Trial Group, Marcela Sarmiento, Anahi Rollheiser, Hugo Ferro, Victoria Caula, Edgardo Garcia Espina, Sebastian Pastoriza, Ross I Baker, Maan Alwan, Peter Thian Guan Tan, Jir‐Ping Boey, Jack Karel Driml, Jennifer Curnow, Emily Blyth, Taryn Freeman, Amanda Johnston, John Kwan, Chun Ma, Jonathan Marwick, Kenneth Micklethwaite, Lachlin Vaughan, Zane Kaplan, Gareth Gregory, Emma Leitinger, Christopher Leow, Alexandra Murton, Jeremy Ong, Ross Salvaris, Maciej Jan Tatarczuch, Jonathan Wong, Ahmad Zargari, Michael Leahy, Philip Young‐Ill Choi, Angeline Audrey Achola Josiah, Kisoth Arasaratnam, Fathima Ayyalil, Samuel Bennett, Philip Crispin, James D’Rozario, Melanie D’Souza, Thomas Gleeson, Maya Latimer, Edwin Lee, Jun Lim, Ross Lindell‐Innes, Mridula Mokoonlall, Jun Ng, Nalini Pati, Michael Pidcock, Kristy Natasha Rady, Caitllin Thirunavukarasu, Pencho Georgiev, Maria Dimitrova, Zhanet Grudeva‐Popova, Bulent Nihat, Alexander M. Arellano Rojas, Christine M. Rojas Hopkins, Eugenia Loredo Fort, Eduardo P. Yáñez Ruiz, Mauro Leandro Morales Sanhueza, Patricio Yanez Weber, Renchi Yang, Yunfei Chen, Ting Sun, Yun Zhuang, Lei Yang, Feng Zhang, Jiajia Li, Jun Li, Lin Liu, Meng Wang, Yanli Yang, Fangbing Zhu, Junfeng Zhu, Heng Mei, Danying Liao, Quizhe Wei, Hu Zhou, Bingjie Ding, Mengjuan Li, Xuewen Song, Lina Zhang, Yuanyuan Zhang, Liang Wang, Jia Cong, Liqiang Wei, Na Yao, Hongyu Zhang, Silang Chen, Jia Feng, Lihua Sun, Haichan Xu, Lei Xu, Qian Zhang, Fengluan Zhong, Guo wei Li, Guoshu Chen, Li Chen, Xiaoyu Chen, Dongting Liu, Xiaohua Qin, Xiaoling Xie, Xiaoming Fei, D. Wenping Lu, Yilong Lu, Ming Luo, Lixia Wang, Xianqiu Yu, Xin Wang, Chao Ding, Xiaoqiong Duan, Wenhua He, Liping Huan, Yanqiu Li, Juan Liu, Ningning Tang, Wei Yin, Mamia Zodelva, Irine Datikashvili‐David, Nino Sharashenidze, Levan Makhaldiani, Teano Gamezardashvili, Ekaterine Gaprindashvili, Tamar Gersamia, Magdana Betaneli, Tamta Makharadze, Anargyros Symeonidis, Maria‐Lamprini Kasti, Alexandra Kourakli, Vasileios Lazaris, Andi Prekazi, Angelos Sarantopoulos, Marina Mantzourani, Vasiliki Antoniadou, Dimitra Vlachopoulou, Antonia Syrigou, Achilles Anagnostopoulos, Eleni Gavriilaki, Michail Iskas, Ioanna Sakellari, Christos Varelas, Ronan G. Desmond, Paraic Behan, Helen Enright, Christopher Fox, John McHugh, Grace Rothwell‐Kelly, Louisa Shackleton, Ilana Levy‐Yurkovski, Natalia Kreiniz, Sarah Matarasso‐Greenfeld, Tamar Tadmor, Anatoly Nemets, Alexander Avroutine, Yosef Barshay, Tatiana Boulygin, Shirley Zafrir‐Haver, Andrea Patriarca, Riccardo Moia, Maura Nocolosi, Wilma Barcellini, Bruno Fattizzo, Juri Giannotta, Felicetto Ferrara, Barbara Pocali, Antonio Cuneo, Maurizio Cavallari, Francesco Cavazzini, Maria Ciccone, Gian Matteo, Mariarosaria Sessa, Paolo Tomasi, Antonio Urso, Fabrizio Pane, Federico Chiurazzi, Giada Muccioli Casadei, Alessandro Lucchesi, Maria Giannini, Giovanni Marconi, Giorgia Micucci, Marianna Norata, Monica Poggiaspalla, Elena Rossi, Valerio De Stefano, Tomoki Ito, Shinya Fujita, Masaaki Hotta, Akiko Konishi, Takahisa Nakanishi, Atsushi Satake, Hideaki Yoshimura, Akio Saito, Naoki Akashi, Hirono Iriuchishima, Akira Matsumoto, Morio Matsumoto, Masahiro Mihara, Keita Nakayama, Morio Sawamura, Yukie Terasaki, Yoshitaka Miyakawa, Ryohei Abe, Atsushi Ohsaki, Kohei Saito, Rie Shiomi, Kodai Suzuki, Kento Umino, Shoko Ito, Masahiro Ashiwaza, Shinichiro Fujiwara, Kaoru Morita, Yoshinobu Kanda, Daisuke Minakata, Rui Murahashi, Hirotomo Nakashima, Ken Ohmine, Tsukasa Ohmori, Kazuya Sato, Yumiko Toda, Masuzu Ueda, Chihiro Yamamoto, Katsutoshi Ozaki, Yuya Kishida, Yasuko Kuribayashi, Keita Kirito, Eriko Hosokawa, Hideto Hyuga, Ichiro Kawashima, Megumi Koshiishi, Takuma Kumagai, Minori Matsuura, Kei Nakajima, Yuma Sakamotot, Masaru Tanaka, Takeo Yamamoto, Ryosuke Ogawa, Kenichi Aoki, Kentaro Kohno, Akihiko Numata, Teppei Obara, Yotaro Tamai, Wataru Kamata, Teiko Kawahigashi, Shuku Sato, Shun Tsunado, Shigeki Ito, Shugo Kowata, Tatsuo Oyake, Suleimman Al‐Sweedan, Dalia Alsweedan, Raghad Irshaid, Nour Sameeh, Muath Thiabat, Abdalla Awidi Abbadi, Feras Al Fararjeh, Alaa Alshorman, Jong‐Ho Won, Kyoung Ha Kim, Seug Yun Yoon, Soo‐Mee Bang, Sang‐A Kim, Jeong‐Ok Lee, Ji Yun Lee, June‐Won Cheong, Ji Eun Jang, Hyun Soo Cho, Haerim Chung, Doh Yu Hwang, Yu Ri Kim, Hyewon Kook, Yunsuk Choi, Kyoo‐Hyung Lee, Eun‐Ji Choi, Je‐Hwan Lee, Jung‐Hee Lee, Hang‐Seung Park, Mariela Cardiel Silva, Ivan Gamez Valdez, Karen Miranda Fernandez, Carlos Chavez Trillo, Alva Zaragoza Vazquez, Marco A. Velasco Rojas, Ximena Arenas Reyes, Jessica Graciela Reyes Contreras, Francisco Hernandez Rodriguez, Kirsty Marshall, Sharon Jackson, Amy J. Holmes, Peter Ganly, Andrew Butler, Wei‐Hsun Hsu, Sean Macpherson, Emma‐Jane McDonald, Wendy Stewart, Waleed Ghanima, Eiriki Tjonnfjord, Sebastian Grosicki, Ewa Bodzenta, Marek Kriegler, Zofia Spyra‐Gorny, Maria Soroka‐Wojtaszko, Oliwia Bachanek‐Mitura, Justyna Kozinska, Szymon Fornagiel, Beata Kumiega, Grzegorz Basak, Jagoda Rytel, Maciej Kazmierczak, Piotr Cieslak, Emil Durka, Marek Kotlarski, Michal Kwiatek, Justyna Pieronkiewicz, Lukasz Pruchniewski, Teresa Melo, Mafalda Alpoim, Margarida Badoir, Henrique Coelho, Cristina Fereira, Marta Nunes, Sofie Ramalheira, Antonio Medina de Almeida, Paulo Bernardo, Paula Braga, Maria Fátima Ferreira, Pedro Afonso Costa Baptista, Marta Henriques, Celina Afonso, Guadalupe Gomes, Francisca Miranda, Filipa Mousinho, Ana Ramalhal Jorge, Mariana Vasilica, Andra Tomescu, Luminita Ocroteala, Gabriela Diana Baluta, Doriana Duta, Ioan Macarie, Marcela Candea, Georgeta Daniela Georgescu, Lidia Mihai, Florina Patrinoiu, Alina Catana, Alice Dumitrescu, Gabriela Borsaru, Anca Ciobanu, Laura Cristina Predescu, Delia‐Monica Dima, Ana Maria Bancos, Daniela Dragancea, Simona Galdean, Nicoleta Negru, Sergey Zakharov, Yulia Dubinina, Alvetina Lidzhieva, Uruzmag Tomaev, I Pospelova Tatiana, Tatiana Babaeva, Olga Seregina, Svetlana A. Volkova, Elena Kiseleva, Valeria Turetskova, Maria Novikova, Zhanna Davydova, Irina Novokreschenova, Svetlana Valerievna Samarina, Marina Kokoreva, Zorica Cvetkovic, Tamara Bibic, Milica Tanasjievic, Andrew B. McDonald, David Brittain, Philippa Ashmore, Karen Gunther, Johnny Mahlangu, Ching Yu Wang, Vernon Johan Louw, Cecile Du Toit, Elzanne Jansen van Rensburg, Gerdlen Johanna Kritzinger, Perry Loebenberg, Gamuchirai Tadzimirwa, Estelle Verburgh, Blanca Sanchez Gonzalez, Sara Garcia Avila, Yao‐yu Hsieh, Yu‐Sheng Chang, Tsu‐Yi Chao, Wei‐Hong Cheng, Po‐Ya Chuang, Sheng‐Hong Lin, Hui‐Wen Liu, Archrob Khuhapinant, Rarinee Rungjirajittranon, Noppacharn Uaprasert, Chatphatai Moonla, Suporn Chuncharunee, Pichika Chantrathammachart, Teeraya Puavilai, Chatree Chai‐Adisaksopha, Pokpong Piriyakhuntorn, Tontanai Numbenjapon, Wittawat Jandamnernpong, Rattapan Lamool, Nonglak Kanitsap, Lantarima Bhoopat, Nattiya Teawtrakul, Kanchana Chansung, Theerin Lanamtieng, Chinadol Wanitpongpun, Abderrahim Khelif, Bechir Achour, Walid Bouteraa, Raihane Ben Lakhal, Yosr Abdnnebi, Dorra Dhaouadi, Emna Maaloul, Moez Elloumi, Moez Medhaffa, Selami Kocak Toprak, Bulent Karakaya, Ekin Kircali, Gul Yavuz, Filiz Vural, Nigar Abdullayeva, Aysenur Arslan, Ajda Gunes, Fatma Keklik Karadag, Tural Pashayev, Denis Sabriye Bozer, Bahar Sevgili, Nur Soyer, Tuba Hacibekiroglu, Yasin Kalpakci, Cenk Sunu, Fevzi Altuntas, Semih Basci, Merih Kizil Cakar, Sema Secilmis, Mehmet Sinan Dal, Bahar Uncu Ulu, Samet Yaman, Tugce Nur Yigenoglu, Anil Tombak, Fatma Akbas, Meliha Nalcaci, Simge Erdem, Dilek Ozden Kubilay, Tarik Tiryaki, Ozgur Mehtap, Ayfer Geduk, Meltem Ayli, Tuba Bulduk, Alparslan Merdin, Melda Ozkan, Selim Sayin, Bilge Ugur, Murat Yildirim, Ahmet Muzaffer Demir, Ufuk Demirici, Elif Umit, Ahmet Yigitbasi, Burhan Turgut, Seval Akpinar, Mehmet Sonmez, Ozlen Bektas, Nergiz Erkut, Benjamin Bailiff, Salama Abosaod, Rabia Afghan, Amy Gudger, Tsan Hei Luk, Timothy Moorby, Maria Mushkbar, Beena Salhan, Elizabeth Tebbet, Adam Forbes, Michale Creagh, Nicole Fowler, Michelle Furtado, Cristina Maria Thiebaud Alvarez, David Tucker, Martha Vickers, Nichola Cooper, Christine Ademokun, Nehal Joshi, Lucy Kamuriwo, Clare Rees, Steven Lentz, Mark Karwal, Usha Perepu, Grerk Sutamtewagul, Catherine M. Broome, Pedro Alcedo, Julia Cunningham, Graig Kessler, Sunil Babu, Farrukh Adhami, Carrie Boots, Shalini Chitneni, Ryan Gonzales, Yasolatha Nalamolu, Sreenivasa Nattam, Guinevere Nilles, Ahad Sadiq, Shelley Seabolt, David Zimmerman, Bashar Alasad, Loralei Floden, Samer Hassan, Nasser Janbay, Kellie Larsen‐Dyer, Eric S. Schaefer, Joseph Beck, Daniel Bradford, Brooke Brander, Lynsay Brautnick, Mikaila Calcagni, K’Anne Cash‐Arthur, Rita Draggs, Diane Glendinning, Cristina Hernandez, Sarah Jewell, Matthew Lockwood, Gregory Oakhill, Stephan Rosenfeld, Patrick Travis +546 morewiley +1 more sourceHepatic Glycogen Storage Diseases in Brazil: A Multicenter Study
American Journal of Medical Genetics Part A, EarlyView.ABSTRACT
To describe clinical and laboratory characteristics, emphasizing the evolution of patients with hepatic glycogen storage diseases (GSDs) followed in Brazilian reference centers. Multicenter, retrospective study involving 13 centers, using RedCap platform. 132 patients were included: 63 (47.8%) GSD type I (56 Ia, 7 Ib), 13 (9.8%) with type III (Mariana Pena Costa, Alexandre Rodrigues Ferreira, Adriana Teixeira Rodrigues, Rodrigo Rezende Arantes, Thais Costa Nascentes Queiroz, Elisa de Carvalho, Gilda Porta, Irene Kazue Miura, Maria Tereza Galvão Guiotti, Rafaella Karen Sousa Monterlei, Adriana Maria Alves de Tommaso, Gabriel Hessel, Maria Ângela Bellomo‐Brandão, Roberta Vacari de Alcantara, Maria Julia Rodrigues Teixeira de Araujo, Daniela Góis Meneses, Regina Sawamura, Cibele Dantas Ferreira Marques, Lucas Rocha Alvarenga, Marise Helena Cardoso Tofoli, Ana Cristina Vieira de Melo, Jussara Melo de Cerqueira Maia, Leticia Helena Caldas Lopes, Eleonora Druve Tavares Fagundes +23 morewiley +1 more sourceAn ultra‐processed food‐based cafeteria diet induces obesity, metabolic dysfunction, and tissue‐specific gene dysregulation in C57BL/6 mice
Animal Models and Experimental Medicine, EarlyView.This schematic figure illustrates the experimental design and key findings of the study. Forty male C57BL/6 mice were assigned to either a standard diet control group or a cafeteria diet (CAFD) group for 16 weeks. CAFD induces obesity, insulin resistance (HOMA‐IR), hepatic steatosis, and dysregulation of a number of genes in visceral and subcutaneous ...Guilherme Coutinho Kullmann Duarte, Daisy Crispim, Shiva Cerutti Wittée, Joana Raquel Nunes Lemos, Natalia Emerim Lemos, Mayara Souza de Oliveira, Melissa Daniele Alves, Eloísa Toscan Massignam, Tais Silveira Assmann, Felipe Mateus Pellenz +9 morewiley +1 more sourceIncorporation of Novel Synthetic Glycolipids in Liposomal Nanoparticles Affects Opsonization and In Vivo Clearance
Angewandte Chemie International Edition, EarlyView.We prepared five glycosylated liposomal nanoparticles (G‐LNPs) to investigate the role of glycosylation and protein corona in modulating the in vivo behavior of G‐LNPs. We show that IgG and complement C3 adsorption enhanced liposomal nanoparticle clearance, with IgG promoting subsequent C3 binding.Yingjie Yu, Xuehan Li, Yu Gao, Shijia Tao, Xiaofei Li, Lemei Zhao, Wenshuai Han, Hao Fan, Ying Qiu, Man Wang, Luying Zhou, Xiaoyan Fang, Wenhua Yang, Haiyang Zhang, Volker Mailänder, Daniel Crespy, Katharina Landfester, Shuai Jiang, Xiangzhao Mao +18 morewiley +1 more source
