Safety, tolerability, and efficacy of subcutaneous efgartigimod in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ADHERE): a multicentre, randomised-withdrawal, double-blind, placebo-controlled, phase 2 trial

Authors: Grinzinger, Susanne, Wanschitz, Julia, Seifert-Held, Thomas, Claeys, Kristl G., Baets, Jonathan, Remiche, Gauthier, Bissay, Véronique, Dubuisson, Nicolas, Delstanche, Stephanie, Tarnev, Ivaylo, Genov, Krasimir, Tsvetanov, Plamen, Milanov, Ivan, Zhao, Chongbo, Bu, Bitao, Yu, Xuefan, Li, Wei, Jiang, Haishan, Da, Yuwei, Lu, Zuneng, Liang, Hui, Guo, Fuqiang, Li, Zunbo, Zou, Zhangyu, Hong, Daojun, Yang, Huan, Guo, Junhong, Shi, Jianquan, Tu, Jianglong, He, Dian, Wang, Yiqi, Ding, Jing, Zhang, Yali, Zhao, Yuanqi, Xu, Renxi, Yue, Yunhua, Guo, Aihong, Wang, Yuzhong, Talab, Radomir, Harbo, Thomas, Sindrup, Soeren, De Seze, Jérôme, Sacconi, Sabrina, Péréon, Yann, Echaniz-Laguna, Andoni, Magy, Laurent, Nicolas, Guillaume, Taithe, Frédéric, Cassereau, Julien, Debs, Rabab, Shakarishvili, Roman, Tsiskaridze, Alexander, Mania, Maka, Janelidze, Marina, Janelidze, Tamar, Schroeter, Michael, Skripuletz, Thomas, Lee, De-Hyung, Klehmet, Juliane, Hotter, Benjamin, Hoffmann, Olaf, Baum, Petra, Zschuentzsch, Jana, Pitarokoili, Kalliopi, Stettner, Mark, Bereczki, Dániel, Pánczél, Gyula, Abraham, Alon, Dori, Amir, Lampl, Yair, Manganelli, Fiore, Morino, Stefania, Padovani, Alessandro, Siciliano, Gabriele, Schenone, Angelo, Magri, Francesca, Mazzeo, Anna, Giannini, Fabio, Sorbi, Sandro, Chiò, Adriano, Kuwahara, Motoi, Okuno, Tatsusada, Okamoto, Tomoko, Kokubun, Norito, Nishiyama, Kazutoshi, Kaida, Kenichi, Bokuda, Kota, Katsuno, Masahisa, Yabe, Ichiro, Saji, Etsuji, Yokota, Takanori, Hatanaka, Yuki, Nakahara, Jin, Sugimoto, Takamichi, Tanaka, Fumiaki, Tomita, Satoshi, Yamano, Yoshihisa, Hayashi, Tomohiro, Yamazaki, Hiroki, Tokashiki, Takashi, Horiuchi, Kazuhiro, Karelis, Guntis, Eftimov, Filip, Banach, Marta, Chyrchel-Paszkiewicz, Urszula, Kochanowicz, Jan, Selmaj, Krzysztof, Zielinski, Tomasz, Banaszkiewicz, Krzysztof, Mitrea, Dan, Scutaru-Kadar, Ana-Maria, Axelerad, Any, Stuchevskaya, Fatima, Boyko, Alexey, Khabirov, Farit, Trushnikova, Tatiana, Goncharova, Zoya, Suponeva, Natalia, Dorogov, Nikolay, Yakupov, Eduard, Raicevic, Ranko, Miletic Drakulic, Svetlana, Cabrera Serrano, Macarena, Muñoz Blanco, Jose Luis, Guerrero Sola, Antonio, Aguera Morales, Eduardo, Diaz Marin, Carmen, Juntas Morales, Raúl, Yeh, Jiann-Horng, Sung, Jia-Ying, Huang, Han-Wei, Tsai, Nai-Wen, Guo, Yuh-Cherng, Chao, Chi-Chao, Ro, Long-Sun, Sengun, Ihsan, Terzi, Murat, Alpaydin Baslo, Sezin, Koç, Filiz, Necdet Karli, Hamdi, Shulga, Olga, Smolko, Dmytro, Doroshenko, Oleksandr, Tomakh, Nataliya, Kyrychenko, Alla, Seliuk, Olga, Kalbus, Oleksandr, Skrypchenko, Iryna, Novakovska, Olha, Carod-Artal, Francisco Javier, Rinaldi, Simon, Brennan, Kathryn, Ellis, Simon, Carr, Aisling, Matthews, Emma, Traub, Rebecca, Mozaffar, Tahseen, Elliott, Matthew, Bhavaraju-Sanka, Ratna, Nance, Christopher, Levine, Todd, Lisak, Robert, Pasnoor, Mamatha, Pulley, Michael, Roy, Bhaskar, Govindarajan, Raghav, Sahagian, Gregory, Khella, Sami, Jacob, Daniel, Kushlaf, Hani, Sivakumar, Kumaraswamy, Melamed, Isaac, Sharma, Khema, Quick, Adam, Ubogu, Eroboghene, Lacomis, David, Isa, Arnaldo, Brannagan, Thomas, Chen, Shan, Katz, Jonathan, Feinberg, Marc, Pavlakis, Pantelis, Lange, Dale, Gudesblatt, Mark, Tandan, Rup, Gable, Karissa, Rivner, Michael, Barnes, Benjamin, Luke, Donna, Mahuwala, Zabeen, Macwan, Samir, Kwon, Patrick, Scott, James, Altamimi, Sadiq, Sabharwal, Priyanka, Allen, Jeffrey A ^{a, *}, Lin, Jie ^b, Basta, Ivana ^c, Dysgaard, Tina ^d, Eggers, Christian ^e, Guptill, Jeffrey T ^{f, g}, Gwathmey, Kelly G ^h, Hewamadduma, Channa ^{i, j}, Hofman, Erik ^f, Hussain, Yessar M ^k, Kuwabara, Satoshi ^l, Le Masson, Gwendal ^m, Leypoldt, Frank ^{n, o}, Chang, Ting ^p, Lipowska, Marta ^{q, r}, Lowe, Murray ^f, Lauria, Giuseppe ^{s, t}, Querol, Luis ^{u, v}, Simu, Mihaela-Adriana ^w, Suresh, Niraja ^x, Tse, Anissa ^f, Ulrichts, Peter ^f, Van Hoorick, Benjamin ^f, Yamasaki, Ryo ^{y, z}, Lewis, Richard A ^{aa, *}, van Doorn, Pieter A ^{ab, *}

Source: In The Lancet Neurology October 2024 23(10):1013-1024

Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

Authors: Vita, Giuseppe, Rizzo, Vincenzo, Russo, Massimo, Mazzeo, Anna, Gentile, Luca, Berk, John L, Brueckner, Caitlin, Lazzari, Victoria, Wiesman, Janice, DeLong, Douglas, Victory, Jennifer, Dalton, James, May, John, Gilmore, Catherine, Attarian, Shahram, Diallo, Saran, Delmont, Emilien, Pouget, Jean, Verschueren, Annie, Grapperon, Aude-Marie, Campana-Salort, Emmanuelle, Conceição, Isabel M, Lopes, Ana, Lamas, Filipa, Neves, Carlos, Castro, Jose, Pereira, Pedro, Castro, Isabel, Franco, Ana, Santos, Miguel Oliveira, de Azevedo Coutinho, Conceição, Falcao de Campos, Catarina, Coelho, Teresa, Hipólito Reis, Antonio, Correia, Nuno, Perez, Javier M, Martins da Silva, Ana, Alves, Cristina, Cardoso, Marcio, Valdrez, Katia, Monte, Julia R, Pessoa, Bernardete, Guimaraes, Nadia, Freitas, Monica, Ramalho, Joana, Ferreira, Natalia, Kuzume, Daisuke, Tard, Celine, Waucquier, Nawal, Rougeaux, Isabelle, Brice, Sylvie, Kasprzyk, Emmanuelle, Elrezzi, Elise, Meguig, Sayah, Hachulla, Eric, Gauvain, Clement, Migaud-Chervy, Maria-Claire, Deplanque, Dominique, Jozefowicz, Elsa, Lebellec, Loic, Adams, David, Balaya-Gouraya, Line, Jehan Lacour, Nathalie, Bournane, Halima, Martin, Nathalie, Elabed, Mongia, Sacko, Niamey, Boubrit, Yasmine, Gaouar, Amina, Rakotondratafika, Fetra, Théaudin-Saliou, Marie, Cauquil-Michon, Cécile, Labeyrie, Celine, Not, Adeline, Al-Salameh, Abdallah, Lecoq, Anne-Lise, Stephant, Maeva, Echaniz-Laguna, Andoni, Becquemont, Laurent, Beaudonnet, Guillemette, Algalarrondo, Vincent, Eliahou, Ludivine, Slama, Michel S, Rousseau, Antoine, Signate, Aissatou, Berthelot, Emeline, Inamo, Jocelyn, Planté-Bordeneuve, Violaine, Vervoitte, Laetitia, Focseneanu, Cecile, Gendre, Thierry, Arrouasse, Raphaele, Ayache, Samar S., Ernande, Laura, Le Corvoisier, Philippe, Salhi, Hayet, Choumert, Ariane, Ehinger, Vincent, Ruiz, Julie, Charlin, Cyril, Megelin, Thomas, Brannagan III, Thomas H, Fayerman, Raisy, Kim, Arreum, Paras, Allan, Gonzalez, Leidy J, Tsang, Steven, Wajnsztajn, Fernanda, Shije, Jeffrey, Ulane, Christina, Kleyman, Inna, Weimer, Louis, Cioroiu, Comana, Lambrianides, Sakis, Abu-Manneh, Rana, Zamba-Papanicolaou, Eleni, Agathangelou, Petros, Leonidou, Eleni, Tada, Satoshi, Fujita, Akemi, Nagai, Masahiro, Ando, Rina, Hosokawa, Yuko, Yamanishi, Yuki, Overcash, J. Scott, Giardino, Elena, Boyer, Leslie, Dang, Lien, Le, An, Nguyen, Tyler, Giang, Lien, Sellers, Peter, Tran, Leyla, Truong, Nghi, Vinas, Maita, Hrkman, Nicole, Miller, Sarah, Nguyen, David, Smith, Ashley, Pu, Helen, Li, Steve, Vuong, Thao, Dioso, Holly, Green, Sinikka, Lee, Kia, Chu, Hanh, Waters, Michael, Coskun, Derya J, Zepeda, Karla A, O'Riordan, William, Obici, Laura, Cortese, Andrea, Lozza, Alessandro, Merlini, Giampaolo, Rosti, Vittorio, Sabatelli, Mario, Bisogni, Giulia, Bernardo, Daniela, Luigetti, Marco, Di Paolantonio, Andrea, Guglielmino, Valeria, Romano, Angela, Nienhuis, Hans, Bulthuis-Kuiper, Janita, Kristen, Arnt V, Gerk, Olga, Ulbricht, Hannah, Taylor, Lenka, Meyle, Eva, Kleinschmidt, Natalia, Meyrath, David, Noe-Schwenn, Simone, Meng, Ulrike, Bauer, Ralf, aus dem Siepen, Fabian, Hein, Selina, Takahashi, Tetsuya, Oshita, Tomohiko, Koujin, Yoko, Neshige, Shuichiro, Nezu, Tomohisa, Segawa, Akiko, Ueno, Hiroki, Morino, Hiroyuki, Campistol, Josep M, Rodas Marin, Lida Maria, Blasco Pelicano, Josep Miquel, Dávila, Lucía Galán, Palacios, Marta, Pytel Cordoba, Vanesa, Guerrero Sola, Antonio, Horga, Alejandro, García Feijoo, Julián, Perez de Isla, Leopoldo, Marques Júnior, Wilson, Moscardini, Mariana, Litcanov, Debora Cristina, Viera Lima, Ana Flavia, Rodrigues, Leonardo, Marques Coutinho, Barbara, Moreira, Carolina Lavigne, Daccach Marques, Vanessa, Munoz Beamud, Francisco, Gragera Martínez, Álvaro, Borrachero, Cristina, Losada López, Inés Asunción, Cisneros Barroso, Eugenia, Rodríguez Rodríguez, Adrián, Sanz, Monica, Rigo Oliver, Elena, González Moreno, Juan, Gamez Martinez, Jose M, Descals, Cristina, Uson, Mercedes, Jose Vega, Francisco, Figuerola, Antoni, Montala, Carles, Waddington-Cruz, Márcia, Dias da Silva, Moises, Gervais de Santa Rosa, Renata, Pinto, Luiz Felipe, Pinto, Marcus Vinicius, Cardoso Berensztejn, Amanda, Barroso, Fabio, Lautre, Andrea, Orellana, Lucas G, González-Duarte Briseño, Maria Alejandra, Cárdenas-Soto, Karla, Jiménez López, Brenda Poled, Pérez-Castañeda, Sandra Lorena, Cantú Brito, Carlos Gerardo, Rivera de la Parra, David, Hernandez Reyes, Jose Pablo, del Mar Saniger Alba, Maria, Criollo Mora, Elia, Parman, Yesim, Rezzan, Kus Jülide, Sahin, Erdi, Serbest, Nail G, Durmus, Hacer, Cakar, Arman, Tugal Tutkun, Nuriye Ilknur, Karamursel, Sacit, Elitok, Ali, Sirin Inan, Nermin G, Altinkurt, Emre, Polydefkis, Michael, Ye, Jing, Allen, Adriane C, Chaudhry, Vinay, Jarrett, Raquel, Bressler, Neil, Burks, Kathleen L, Liu, Qingfeng, Khoshnoodi, Mohammad, Judge, Daniel P, Vista, Geno, Shah, Syed Mahmood, Hamaguchi, Hirotoshi, Oda, Junko, Fukase, Emi, Taniguchi, Ikuko, Oda, Tetsuya, Endo, Hironobu, Shimomura, Masahiro, Katanazaka, Kimitaka, Koto, Shusuke, Nakano, Takahiro, Scheid, Christof, Zueiter, Andreas, Pester, Lars, Walter, Doreen, Özdemir, Betül, Frenzel, Lukas F, Holtick, Udo, Oh, Jeeyoung, Kim, Hee Jin, Shin, Hyun Jin, Choi, Kyomin, Yamashita, Taro, Ueda, Mitsuharu, Masuda, Teruaki, Misumi, Yohei, Ueda, Akihiko, Nakahara, Keiichi, Yorita, Akiko, Tsuruhisa, Seiko, Taniwaki, Takayuki, Harada, Masaya, Moritaka, Taiga, Sakurada, Naonori, Mauricio, Elizabeth A, Baskin, Amber, Dimberg, Elliot, Dispenzieri, Angela, Fonder, Amie, Hobbs, Miriam, Russell, Stephen J, Dyck, Peter, Gonsalves, Wilson, Leung, Nelson, Witzig, Thomas E, Zeldenrust, Steven R, Hwa, Lisa, Kapoor, Prashant, Kumar, Shaji K, Lin, Yi, Lust, John A, Rajkumar, Vincent S, Dingli, David, Gertz, Morie A, Go, Ronald, Hayman, Suzanne R, Dalia, Samir, Carrillo, Esmeralda, Gorevic, Peter, Mason, Garnette, Chao, Chi-Chao, Lee, Ming-Jen, Su, Jen-Jen, Hsieh, Sung-Tsang, Tsai, Li-Kai, Yeh, Shin-Joe, Yang, Chih-Chao, Ajroud-Driss, Senda Ajroud-Driss, Casey, Patricia, Joslin, Benjamin C, Freimer, Miriam, Sankey, Alison, Kenepp, Amanda, Heintzman, Sarah, LoRusso, Samantha, Hokezu, Youichi, Kim, Byoung-Joon, Kim, JuHyeon, Lee, Ga Yeon, Cho, Eun Bin, Jeon, Eun-Seok, Min, Ju-Hong, Seok, Jin Myoung, Lee, Hye Lim, Park, Jae Hong, Sekijima, Yoshiki, Miyazawa, Chinatsu, Kato, Nagaaki, Kishida, Dai, Hineno, Akiyo, Kodaira, Minori, Yoshinaga, Tsuneaki, Miyahara, Teruyoshi, Imai, Akira, Matsumoto, Kazuhiko, Lin, Kon-Ping, Lee, Yi-Chung, Wixner, Jonas, Falk, Malin, Pilebro, Bjorn, Suhr, Ole, Lindqvist, Per, Soderberg, Karin, Pedrosa-Domellöf, Fatima, Anan, Intissar, Nordh, Erik, Tournev, Ivaylo, Zhelyazkova-Glaveeva, Sashka, Cherneva, Zheyna, Sarafov, Staiko, Chamova, Teodora, Cherninkova-Gopina, Sylvia, Schmidt, Hartmut H, Friebel, Frauke, Zibert, Andree, Mihailovic, Natasa, Schubert, Friederike, Vorona, Elena, Lahme, Larissa, Huesing-Kabar, Anna, Schilling, Matthias, Kabar, Iyad, Gillmore, Julian D, Martinez-Naharro, Ana, Chacko, Liza, Cohen, Oliver, Law, Steven, Rezk, Tamer, Lachmann, Helen J, Quan, Dianna, Blume, Brianna, Dixon, Stacy, Low, Soon Chai, Chan, Soo Looi, Lim, He Eng Li, Goh, Khean Jin, Mezei, Michelle M, Kraus, Deborah, Jack, Kristin, Wade, N. Kevin, Lopate, Glenn, Zwijack, Brittany, Florence, Julaine, Sommerville, R. Brian, Stewart, Graeme, Ryder, Julie, Mekhael, Linda, Taylor, Mark, Suan, Daniel, Wells, Karen, Stone, Paula, Itoya, Amenze, Owusu-Sekyere, Mercy, Thai, Desmond, Chahine, Ilonah, Pedrosa, Salve, Do, Thi Hoa (Therese), González-Duarte, Alejandra, Kyriakides, Theodoros, Ajroud-Driss, Senda, Mauricio, Elizabeth, Brannagan, Thomas H, III, Aldinc, Emre, Wang, Jing Jing, White, Matthew T, Vest, John, Berber, Erhan, Sweetser, Marianne T

Source: In The Lancet Neurology January 2021 20(1):49-59

Lipid Metabolic Alterations in the ALS–FTD Spectrum of Disorders.

Authors: Godoy-Corchuelo, Juan Miguel, Fernández-Beltrán, Luis C., Ali, Zeinab, Gil-Moreno, María J., López-Carbonero, Juan I., Guerrero-Sola, Antonio, Larrad-Sainz, Angélica, Matias-Guiu, Jorge, Matias-Guiu, Jordi A., Cunningham, Thomas J., Corrochano, Silvia

Source: Biomedicines; May2022, Vol. 10 Issue 5, p1105, 23p

Subject Terms: AMYOTROPHIC lateral sclerosis, LIPID metabolism disorders, FRONTOTEMPORAL dementia, LIPIDS, LIPIDOMICS

Drug‐refractory myasthenia gravis: Clinical characteristics, treatments, and outcome.

Authors: Cortés‐Vicente, Elena, Álvarez‐Velasco, Rodrigo, Pla‐Junca, Francesc, Rojas‐Garcia, Ricard, Paradas, Carmen, Sevilla, Teresa, Casasnovas, Carlos, Gómez‐Caravaca, María Teresa, Pardo, Julio, Ramos‐Fransi, Alba, Pelayo‐Negro, Ana Lara, Gutiérrez‐Gutiérrez, Gerardo, Turon‐Sans, Janina, López de Munain, Adolfo, Guerrero‐Sola, Antonio, Jericó, Ivonne, Martín, María Asunción, Mendoza, María Dolores, Morís, Germán, Vélez‐Gómez, Beatriz

Source: Annals of Clinical & Translational Neurology; Feb2022, Vol. 9 Issue 2, p122-131, 10p

Subject Terms: MYASTHENIA gravis, PROTEIN-tyrosine kinases, DRUG toxicity, IMMUNOSUPPRESSIVE agents

Geographic Terms: AMERICA

Clinical characteristics and outcomes of thymoma‐associated myasthenia gravis.

Authors: Álvarez‐Velasco, Rodrigo, Gutiérrez‐Gutiérrez, Gerardo, Trujillo, Juan Carlos, Martínez, Elisabeth, Segovia, Sonia, Arribas‐Velasco, Marina, Fernández, Guillermo, Paradas, Carmen, Vélez‐Gómez, Beatriz, Casasnovas, Carlos, Nedkova, Velina, Guerrero‐Sola, Antonio, Ramos‐Fransi, Alba, Martínez‐Piñeiro, Alicia, Pardo, Julio, Sevilla, Teresa, Gómez‐Caravaca, María Teresa, López de Munain, Adolfo, Jericó, Ivonne, Pelayo‐Negro, Ana L.

Source: European Journal of Neurology; Jun2021, Vol. 28 Issue 6, p2083-2091, 9p

Subject Terms: THYMOMA, MYASTHENIA gravis, TREATMENT effectiveness, RECEPTOR antibodies, PROGNOSIS, SYMPTOMS

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Clinical and therapeutic features of myasthenia gravis in adults based on age at onset.

Authors: Cortés-Vicente, Elena, Álvarez-Velasco, Rodrigo, Segovia, Sonia, Paradas, Carmen, Casasnovas, Carlos, Guerrero-Sola, Antonio, Pardo, Julio, Ramos-Fransi, Alba, Sevilla, Teresa, López de Munain, Adolfo, Gómez, Maria Teresa, Jericó, Ivonne, Gutiérrez-Gutiérrez, Gerardo, Pelayo-Negro, Ana Lara, Asunción Martín, María, Mendoza, María Dolores, Morís, Germán, Rojas-Garcia, Ricard, Díaz-Manera, Jordi, Querol, Luis

Source: Neurology; 3/17/2020, Vol. 94 Issue 11, pe1171-e1180, 10p

Notch Signalling in the Hippocampus of Patients With Motor Neuron Disease.

Authors: Gómez-Pinedo, Ulises, Galán, Lucía, Matías-Guiu, Jordi A., Pytel, Vanesa, Moreno, Teresa, Guerrero-Sola, Antonio, Matías-Guiu, Jorge

Source: Frontiers in Neuroscience; 4/5/2019, pN.PAG-N.PAG, 13p

Subject Terms: HIPPOCAMPUS (Brain), NOTCH signaling pathway, MOTOR neuron diseases, DEVELOPMENTAL neurobiology, BIOLOGICAL tags

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Subventricular zone in motor neuron disease with frontotemporal dementia

Authors: Galan, Lucia, Gomez-Pinedo, Ulises, Vela-Souto, Alvaro, Guerrero-Sola, Antonio, Barcia, Juan Antonio, Gutierrez, Alberto Rábano, Martinez-Martinez, Armando, Jiménez, Maria Sagrario Barquero, García-Verdugo, Jose Manuel, Matias-Guiu, Jorge

Source: In Neuroscience Letters 2011 499(1):9-13

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