Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial

Autor: Lucy Lam, Jiri Pitha, John Vissing, Laura Fionda, Denis Korobko, Michael Pulley, Tony Vangeneugden, Silvia Bonanno, Nobuhiro Ido, Jerrica Farias, Jafar Shabanpour, James Gilchrist, Girolamo Alfieri, Gyorgyi Szabo, Carlayne E. Jackson, Eduardo Ng, Csilla Rozsa, Hans D. Katzberg, Carlo Antozzi, Aleksandra Golenia, Sayaka Ishida, Temur Margania, Andrzej Szczudlik, Richard J. Barohn, Mari Suzuki., Robert Henegar, Kaoru Sakuma, Evanthia Bernitsas, Elena Lapochka, Yukiko Ozawa, Tomihiro Imai, Debbie Hastings, Antonio Guglietta, Benjamin Frishberg, Elena Antipenko, Vera Bril, Stanislav Vohanka, Isela Hernandez, Ivo Bozovic, Ilona Vergunova, Lorenzo Maggi, Andreas Meisel, Ali Malekniazi, Tahseen Mozaffar, Emmanuelle Salort-Campana, Yasmin Camberos, Jan J.G.M. Verschuuren, Masayuki Masuda, Masanori Takahashi, Yoshihiko Okubo, Marek Smilowski, Yasushi Suzuki, Mary Wagoner, Andrew Heim, David Bors, Chiho Watanabe, Fiammetta Vanoli, Antonio Reia, Zubair Quraishi, Omar Jawdat, Makoto Samukawa, Gregory Sahagian, Gedeonne Margo Jakab, Eiichi Nomura, Samantha Colgan, Cindy Benzel, Ayako Mori, Annabel M. Ruiter, Ratna Bharavaju-Sanka, Roman Shakarishvili, Victoria Cannon, Malkova Nadezhda, Tomas Horak, Anna Kostera-Pruszczyk, Eniko Szabo, Emilien Delmont, Alexander Tsiskaridze, Lubna Daniyal, Vidosava Rakocevic Stojanovic, Szilvia Toth, Siegfried Kohler, Iveta Novakova, Katherine Roath, Kazuna Ikeda, Salma Akhter, Claudia Heibutzki, Martijn R. Tannemaat, Marta Pinkosz, Mads Peter Godtfeldt Stemmerik, Chafic Karam, Irys Caristo, Carolyn Paiz, Josef Bednarik, Monika Frasinska, Stefania Morino, Norianne Pimentel, Kanako Minemoto, Rekha Pillai, Linda Wagemaekers, Annelien De Pue, Irina Poverennova, Katerina Reguliova, Jana Junkerova, Angela Marsili, Anne-Marie Peters, Maren Wyckmans, Michaela Tyblova, Debbie Eggleston, Anne Mette Ostergaard Autzen, Takamichi Sugimoto, Kuldeep Kumar Khatri, Niraja Suresh, Jan De Bleecker, Lea Gerischer, Grazyna Zwolinska, Kevin R Keene, Yosuke Onishi, Francesco Saccà, Zaeem A. Siddiqi, Marjolein Van Heur, Jeffrey Statland, Tatiana Romanova, Diana Dimitrova, Stojan Peric, Tomoko Tsuda, Cathy Bailey, Lubov Urtaeva, Lizzie Zafirakos, Katherine Ruzhansky, Tomoya Kubota, Angela Campanella, Nadezhda Kuznetsova, Sarah Jones, Giovanni Antonini, Hiroyuki Murai, Luca Leonardi, Alan R. Berger, Jonathan Baets, Peter Ulrichts, Said R. Beydoun, Michala Jakubikova, Mamatha Pasnoor, James F. Howard, Leila Darki, Katerina Havelkova, Namita Goyal, Akiyuki Uzawa, Tia Nguyen, Miki Takaki, Matteo Garibaldi, Manisha Kak, Ivonne Turner, Aude-Marie Grapperon, Mageda Horakova, Yuebing Li, Ivana Basta, Lech Szczechowski, Shabber Mannan, Aneta Pasko, Caroline Vinck, Riccardo Giossi, Rudolf Mercelis, Ivana Jurajdova, Lesly Welsh, Małgorzata Bilińska, Marek Halas, Dragana Lavrnic, Kimiaki Utsugisawa, Todd Levine, Erik Velasquez, Daisuke Yamamoto, Constantine Farmakidis, John Anthony Morren, Sarah Hoffman, Manisha Chopra, Shingo Konno, Rita Frangiamore, Kelly Jia, Jana Horakova, Anna Melnikova, Piotr Szczudlik, Ali Habib, Giorgia Puorro, Michael D. Weiss, Robert P. Lisak, Hiroyuki Naito, Shahram Attarian, Hiroko Nakamura, Shin Hisahara, Mazen M. Dimachkie, Genya Watanabe, Duaa Jabari, Ekaterina Bulatova, Angela Genge, Makiko Naito, Melissa Currence, Henning Andersen, Katrien De Mey, Kathy de Koning, Yuen T. So, Chiara Pane, Renato Mantegazza, Rebecca Traub, Manato Yasuda, Amy Visser, Dike Remstedt, Yuka Takematsu, Frauke Stascheit, Ayumi Kamei, Tuan Vu, Tulio E. Bertorini, Ludivine Kouton, Neelam Goyal, Flicia Mada, Nizar Chahin, Mihiro Shimizu, Srikanth Muppidi, Erina Sugano

Publikováno v: ADAPT Investigator Study Group 2021, ' Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT) : a multicentre, randomised, placebo-controlled, phase 3 trial ', The Lancet Neurology, vol. 20, no. 7, pp. 526-536 . https://doi.org/10.1016/S1474-4422(21)00159-9
The lancet neurology

Background: There is an unmet need for treatment options for generalised myasthenia gravis that are effective, targeted, well tolerated, and can be used in a broad population of patients. We aimed to assess the safety and efficacy of efgartigimod (AR

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2be57499b1a7a7d2e60c700dc07fa4ee
https://pubmed.ncbi.nlm.nih.gov/34426685

Long-term effect of thymectomy plus prednisone versus prednisone alone in patients with non-thymomatous myasthenia gravis: 2-year extension of the MGTX randomised trial

Autor: Gil I Wolfe, Henry J Kaminski, Inmaculada B Aban, Greg Minisman, Hui-Chien Kuo, Alexander Marx, Philipp Ströbel, Claudio Mazia, Joel Oger, J Gabriel Cea, Jeannine M Heckmann, Amelia Evoli, Wilfred Nix, Emma Ciafaloni, Giovanni Antonini, Rawiphan Witoonpanich, John O King, Said R Beydoun, Colin H Chalk, Alexandru C Barboi, Anthony A Amato, Aziz I Shaibani, Bashar Katirji, Bryan R F Lecky, Camilla Buckley, Angela Vincent, Elza Dias-Tosta, Hiroaki Yoshikawa, Márcia Waddington-Cruz, Michael T Pulley, Michael H Rivner, Anna Kostera-Pruszczyk, Robert M Pascuzzi, Carlayne E Jackson, Jan J G M Verschuuren, Janice M Massey, John T Kissel, Lineu C Werneck, Michael Benatar, Richard J Barohn, Rup Tandan, Tahseen Mozaffar, Nicholas J Silvestri, Robin Conwit, Joshua R Sonett, Alfred Jaretzki, John Newsom-Davis, Gary R Cutter, Gary Cutter, Inmaculada Aban, Michelle Feese, Gil Wolfe, Henry Kaminski, Joshua Sonett, Valeria Saluto, Moises Rosenberg, Valeria Alvarez, Lisa Rey, John King, Helmut Butzkueven, John Goldblatt, John Carey, John Pollard, Stephen Reddel, Nicholas Handel, Brian McCaughan, Linda Pallot, Ricardo Novis, Carlos Boasquevisque, Rubens Morato-Fernandez, Manoel Ximenes, Lineu Werneck, Rosana Scola, Paulo Soltoski, Colin Chalk, Fraser Moore, David Mulder, Lisa Wadup, Michele Mezei, Kenneth Evans, Theresa Jiwa, Anne Schaffar, Chris White, Cory Toth, Gary Gelfand, Susan Wood, Elizabeth Pringle, Jocelyn Zwicker, Donna Maziak, Farid Shamji, Sudhir Sundaresan, Andrew Seely, Gabriel Cea, Renato Verduga, Alberto Aguayo, Sebastian Jander, Philipp Zickler, Michael Klein, Cleo-Aron Weis, Arthur Melms, Felix Bischof, Hermann Aebert, Gerhard Ziemer, Björn Thümler, Thomas Wilhem-Schwenkmezger, Eckhard Mayer, Berthold Schalke, Peter Pöschel, Gisela Hieber, Karsten Wiebe, Alessandro Clemenzi, Vanessa Ceschin, Erino Rendina, Federico Venuta, Stefania Morino, Elisabetta Bucci, Luca Durelli, Alessia Tavella, Marinella Clerico, Giulia Contessa, Piero Borasio, Serenella Servidei, Pierluigi Granone, Renato Mantegazza, Emilia Berta, Lorenzo Novellino, Luisa Spinelli, Masakatsu Motomura, Hidenori Matsuo, Takeshi Nagayasu, Masaharu Takamori, Makoto Oda, Isao Matsumoto, Yutaka Furukawa, Daisuke Noto, Yuko Motozaki, Kazuo Iwasa, Daisuke Yanase, Guillermo Garcia Ramos, Bernardo Cacho, Lorenzo de la Garza, Anne Kostera-Pruszczyk, Marta Lipowska, Hubert Kwiecinski, Anna Potulska-Chromik, Tadeusz Orlowski, Ana Silva, Marta Feijo, António Freitas, Jeannine Heckmann, Andrew Frost, Edward Pan, Lawrence Tucker, Johan Rossouw, Fiona Drummond, Isabel Illa, Jorge Diaz, Carlos Leon, Jiann-Horng Yeh, Hou-Chang Chiu, Yei-San Hsieh, Supoch Tunlayadechanont, Sukasom Attanavanich, Jan Verschuuren, Chiara Straathof, Maarten Titulaer, Michel Versteegh, Arda Pels, Yvonne Krum, M. Isabel Leite, David Hilton-Jones, Chandi Ratnatunga, Maria Farrugia, Richard Petty, James Overell, Alan Kirk, Andrew Gibson, Chris McDermott, David Hopkinson, Bryan Lecky, David Watling, Dot Marshall, Sam Saminaden, Deborah Davies, Charlotte Dougan, Siva Sathasivam, Richard Page, Jon Sussman, John Ealing, Peter Krysiak, Anthony Amato, Mohammad Salajegheh, Michael Jaklitsch, Kristen Roe, Tetsuo Ashizawa, Robert Glenn Smith, Joseph Zwischenberg, Penny Stanton, Alexandru Barboi, Safwan Jaradeh, William Tisol, Mario Gasparri, George Haasler, Mary Yellick, Cedric Dennis, Richard Barohn, Mamatha Pasnoor, Mazen Dimachkie, April McVey, Gary Gronseth, Arthur Dick, Jeffrey Kramer, Melissa Currence, Laura Herbelin, Jerry Belsh, George Li, John Langenfeld, Mary Ann Mertz, Taylor Harrison, Seth Force, Sharon Usher, Said Beydoun, Frank Lin, Steve DeMeester, Salem Akhter, Ali Malekniazi, Gina Avenido, Brian Crum, Margherita Milone, Stephen Cassivi, Janet Fisher, Chad Heatwole, Thomas Watson, James Hilbert, Alexis Smirnow, B. Jane Distad, Michael Weiss, Douglas Wood, Joanna Haug, Raina Ernstoff, Jingyang Cao, Gary Chmielewski, Robert Welsh, Robin Duris, Laurie Gutmann, Gauri Pawar, Geoffrey Marc Graeber, Patricia Altemus, Christopher Nance, Ludwig Gutmann, Carlayne Jackson, Patrick Grogan, John Calhoon, Pamela Kittrell, Deborah Myers, Ghazala Hayat, Keith Naunheim, Susan Eller, Eve Holzemer, Amer Alshekhlee, Jason Robke, Brenda Karlinchak, Jonathan Katz, Robert Miller, Ralph Roan, Dallas Forshew, John Kissel, Bakri Elsheikh, Patrick Ross, Sharon Chelnick, Richard Lewis, Agnes Acsadi, Frank Baciewicz, Stacey Masse, Janice Massey, Vern Juel, Mark Onaitis, James Lowe, Bernadette Lipscomb, Gaby Thai, Jeffrey Milliken, Veronica Martin, Ronnie Karayan, Suraj Muley, Gareth Parry, Sara Shumway, Shin Oh, Gwen Claussen, Liang Lu, Robert Cerfolio, Angela Young, Marla Morgan, Robert Pascuzzi, John Kincaid, Kenneth Kesler, Sandy Guingrich, Angi Michaels, Lawrence Phillips, Ted Burns, David Jones, Cindy Fischer, Michael Pulley, Alan Berger, Harry D'Agostino, Lisa Smith, Michael Rivner, Jerry Pruitt, Kevin Landolfo, Demetric Hillman, Aziz Shaibani, Angelo Sermas, Ross Ruel, Farah Ismail, Mark Sivak, Martin Goldstein, Jorge Camunas, Joan Bratton, Hill Panitch, Bruce Leavitt, Marilee Jones, Srikanth Muppidi, Steven Vernino, Sharon Nations, Dan Meyer, Nina Gorham

Publikováno v: The Lancet. Neurology, vol 18, iss 3
The Lancet Neurology, 18(3), 259-268

Summary Background The Thymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone (MGTX) showed that thymectomy combined with prednisone was superior to prednisone alone in improving clinical status as measured by the Quantit

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::da06c81f83043b79520e4f7b276cc0ef
https://hdl.handle.net/1887/120808

An instrumented timed up and go in facioscapulohumeral muscular dystrophy

Autor: Jessie, Huisinga, Adam, Bruetsch, Ayla, Mccalley, Melissa, Currence, Laura, Herbelin, Omar, Jawdat, Mamatha, Pasnoor, Mazen, Dimachkie, Richard, Barohn, Jeffrey, Statland

Publikováno v: Musclenerve. 57(3)

INTRODUCTION: Instrumenting timed functional motor tasks may reveal a continuum of motor disability that predicts future motor dysfunction. METHODS: We performed a prospective study of the instrumented timed up and go (iTUG) test in genetically confi

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::7d1fd12d44150ca44cb22eb82fb37788
https://pubmed.ncbi.nlm.nih.gov/28877559

Corrigendum.

Publikováno v: Muscle & Nerve; Oct2019, Vol. 60 Issue 4, p484-484, 1p

Randomized phase 2 study of ACE‐083, a muscle‐promoting agent, in facioscapulohumeral muscular dystrophy.

Autor: Statland, Jeffrey M., Campbell, Craig, Desai, Urvi, Karam, Chafic, Díaz‐Manera, Jordi, Guptill, Jeffrey T., Korngut, Lawrence, Genge, Angela, Tawil, Rabi N., Elman, Lauren, Joyce, Nanette C., Wagner, Kathryn R., Manousakis, Georgios, Amato, Anthony A., Butterfield, Russell J., Shieh, Perry B., Wicklund, Matthew, Gamez, Josep, Bodkin, Cynthia, Pestronk, Alan

Publikováno v: Muscle & Nerve; Jul2022, Vol. 66 Issue 1, p50-62, 13p