The sense of antisense therapies in ALS
Van Daele, Sien H., Masrori, Pegah, Van Damme, Philip, Van Den Bosch, Ludo
Published in Trends in molecular medicine (01.03.2024)
Published in Trends in molecular medicine (01.03.2024)
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RNF170 mutation causes autosomal dominant sensory ataxia with variable pyramidal involvement
Van Daele, Sien H., Moisse, Matthieu, Race, Valérie, Van Eesbeeck, Amélie, Keldermans, Liesbeth, Vermeer, Sascha, Van Esch, Hilde, Claeys, Kristl G., Van Damme, Philip
Published in European journal of neurology (01.01.2022)
Published in European journal of neurology (01.01.2022)
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A novel heterozygous likely pathogenic SLC5A2 variant in a diabetic patient with glucosuria and aminoaciduria
Inthasot, Saohoine, Vanderhulst, Julien, Janssens, Peter, Van Daele, Sien, Van Hoof, Evelien, Kint, Cyrielle, Iconaru, Laura, de Filette, Jeroen
Published in Endocrinology, diabetes & metabolism case reports (01.07.2024)
Published in Endocrinology, diabetes & metabolism case reports (01.07.2024)
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Diagnostic yield of testing for RFC1 repeat expansions in patients with unexplained adult-onset cerebellar ataxia
Van Daele, Sien Hilde, Vermeer, Sascha, Van Eesbeeck, Amélie, Lannoo, Laura, Race, Valérie, van Damme, Philip, Claeys, Kristl, Vandenberghe, Wim
Published in Journal of neurology, neurosurgery and psychiatry (01.11.2020)
Published in Journal of neurology, neurosurgery and psychiatry (01.11.2020)
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Long‐term safety and efficacy of eculizumab in generalized myasthenia gravis
Illa, Isabel, Wang, Jing Jing, Garcia, Alejandra Dalila, de Koning, Kathy, Stevens, Olivier, Pozo, Marina, Souza Duca, Luciana, Guerreiro, Alexandre, Carrara, Fernanda, Siddiqi, Zaeem, Kurkova, Barbora, Pitha, Jiri, Wolfova, Marcela, Pedersen, Jane, Oksaranta, Olli, Jakab, Gedeonne, Antonini, Giovanni, Di Pasquale, Antonella, Costabile, Teresa, Marsili, Angela, Bonanno, Silvia, Camera, Giorgia, Pasanisi, Maria, Gabriele, Maria, Uzawa, Akiyuki, Matsushita, Takuya, Kikutake, Keiko, Nakamura, Yumiko, Samukawa, Makoto, Mitazaki, Teiichiro, Yoshimura, Shunsuke, Tsurumaru, Masako, Makamori, Masayuki, Takahashi, Masanori, Hasuike, Tomoya, Kobayashi, Hisako, Osakada, Kaori, Imai, Tomihiro, Tsuda, Emiko, Murahara, Takashi, Suzuki, Shuichiro, Uwasa, Kaori, Min, Ju-Hong, Koh, Da Yoon, Kwon, JuYoung, Hwang, Ji Ye, Kim, Miri, Jung, JinWoo, Shin, Ha Young, Shin, Miju, Lazaro, Ana, Diez Tejedor, Exuperio, Salvado, Maria, Mazuela, Gonzalo, Diaz-Manera, Jordi, Piehl, Fredrik, Hietala, Albert, Sengun, Ihsan, Bekircan‐Kurt, Can Ebru, Orsel, Gulsah, Semiz, Cansu, Veske, Adife, Gollogly, Jakit, Sokratous, Giannis, Sohail, Faisal, Arndt, Heike, Amato, Anthony, Salajegheh, Mohammed, Patrick, Kara, Bravver, Elena, Cunningham, Scott, Schommer, Clara, Carnes, Donna, Bodkin, Cynthia, Carter, Cynthia, Jeffery, Dennis, Chelnick, Sharon, Marburger, Tessa, Gilchrist, James, Andoin, Angela, Seelback, Jenna, Bhavaraju-Sanka, Ratna, Alsharabati, Mohammad, Lu, Liang, Rosenfeld, Jeffrey, Pulley, Michael, Shah, Jaimin, Gutmann, Ludwig, Pasnoor, Mamatha, Jenci, Nicole, Locheke, Jerry, Fondaw, Alex, Sharma, Khema, Steele, Julie, Chopra, Manisha, Katzin, Lara, Beydoun, Said, Waheed, Waqar, Distad, Jane, Robeson, Kimberly
Published in Muscle & nerve (01.07.2019)
Published in Muscle & nerve (01.07.2019)
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Efficacy and Safety of Rozanolixizumab in Moderate to Severe Generalized Myasthenia Gravis: A Phase 2 Randomized Control Trial
Bril, Vera, Benatar, Michael, Andersen, Henning, Vissing, John, Brock, Melissa, Greve, Bernhard, Kiessling, Peter, Woltering, Franz, Griffin, Laura, Van den Bergh, Peter
Published in Neurology (09.02.2021)
Published in Neurology (09.02.2021)
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Journal Article
Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension
Mantegazza, Renato, Wolfe, Gil I, Muppidi, Srikanth, Wiendl, Heinz, Fujita, Kenji P, O'Brien, Fanny L, Booth, Heather D E, Howard, Jr, James F
Published in Neurology (26.01.2021)
Published in Neurology (26.01.2021)
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Journal Article
Genetic variability in sporadic amyotrophic lateral sclerosis
Van Daele, Sien Hilde, Moisse, Matthieu, van Vugt, Joke J F A, Zwamborn, Ramona A J, van der Spek, Rick, van Rheenen, Wouter, Van Eijk, Kristel, Kenna, Kevin, Corcia, Philippe, Vourc'h, Patrick, Couratier, Philippe, Hardiman, Orla, McLaughin, Russell, Gotkine, Marc, Drory, Vivian, Ticozzi, Nicola, Silani, Vincenzo, Ratti, Antonia, de Carvalho, Mamede, Mora Pardina, Jesús S, Povedano, Monica, Andersen, Peter M, Weber, Markus, Başak, Nazli A, Shaw, Chris, Shaw, Pamela J, Morrison, Karen E, Landers, John E, Glass, Jonathan D, van Es, Michael A, van den Berg, Leonard H, Al-Chalabi, Ammar, Veldink, Jan, Van Damme, Philip
Published in Brain (London, England : 1878) (01.09.2023)
Published in Brain (London, England : 1878) (01.09.2023)
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Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial
Benatar, Michael, Hansen, Thomas, Geist, Marie A, Blaettler, Thomas, van den Berg, Leonard H, Morales, Raul Juntas, Chio, Adriano, Andersen, Peter M, Pradat, Pierre-Francois, Lange, Dale, Van Damme, Philip, Mora, Gabriele, Grudniak, Mariusz, Olney, Nicholas, Ladha, Shafeeq, Goyal, Namita A, Meyer, Thomas, Hanna, Michael G, Quinn, Colin, Zinman, Lorne, Jabari, Duaa, Shoesmith, Christen, Ludolph, Albert C, Neuwirth, Christoph, Nations, Sharon, Shefner, Jeremy M, Turner, Martin R, Wuu, Joanne, Bennett, Richard, Dang, Hoang, Sundgreen, Claus, Granit, Volkan, Steele, Julie, Levy, Wendy, Hernandez, Jessica, Bilsker, Martin, Jablońska, Dorota, Janse van Mantgem, Mark R, Bunte, Tommy M, Broere, Bianca, de Fockert, Arianne, Sanchez-Tejerina, Daniel, Calvo, Andrea, Moglia, Cristina, Manera, Umberto, Canosa, Antonio, Vasta, Rosario, Salamone, Paolina, Fuda, Giuseppe, Casale, Federico, Winroth, Ivar, Almgren Stenberg, Erica, Holmgren, Monica, Lenglet, Timothee, Querin, Giorgia, Pavlakis, Pantelis, Holzberg, Shara, Sideri, Riccardo, Marinou, Kalliopi, Czarnecki, Maciej, Ługiewicz, Renata, Biel-Czarnecka, Marta, Boczkowska, Marcelina, Schotte, Caroline, Vynckier, Jan, Claeys, Thomas, Delmotte, Koen, Swinnen, Bart, Serrien, Anouk, D'Hondt, Ann, Lamaire, Nikita, Debien, Elisa, Jones, Sarah, Vachon, Chris, Grogan, James, Solorzano, Guillermo, Rakocevic, Goran, Wagoner, Mary, Flavia, Wiehler, Sonja, Körner, Olivia, Schreiber-Katz, Camilla, Wohnrade, Anastasia, Sarikidi, Chantal, Fischer, Adamo, Ashley, Turcotte, Nicole, Duncan, Jessie, Massie, Rami, Berube, Maxime, Saunders, Natalie, Salmon, Kristiana, Foucher, Juliette, Agessandro, Abrahao, Phung, Liane, Jawdat, Omar, Farmakidis, Constantine, Lillig, Katie, Lackey, Alyssa, Weber, Markus, Levine, Todd
Published in Lancet neurology (01.07.2024)
Published in Lancet neurology (01.07.2024)
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Journal Article
Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study
Nowak, Richard J., Mozaffar, Tahseen, Wilken, Miguel, Saba, Juliet, Bettini, Mariela, De Bleecker, Jan, Van den Abeele, Guy, Mahieu, Délphine, Stevens, Olivier, Geraldo, Jordana Gonçalves, Pozo, Marina, Volpe, Luciana Renata Cubas, Ferreira, Luciana, Cassia Santos, Aline, Andrade, Ana Carolina Amaral, Carrara, Fernanda, Junkerova, Jana, Krogh, Susanne, Autzen, Anne Mette Ostergaard, Erälinna, Juha‐Pekka, Oksaranta, Olli, Szabadosne, Brigitta, Antonini, Giovanni, Puorro, Giorgia, Lopergolo, Diego, Onesti, Emanuela, Frasca, Vittorio, Kanzaki, Akiko, Shimpo, Misa, Morikawa, Miyuki, Shiraishi, Hirokazu, Asada, Shizuka, Amamoto, Shoko, Takada, Kazumi, Suzuki, Yasushi, Akiyama, Tetsuya, Narikawa, Koichi, Fukuda, Yusuke, Sato, Miwako, Nakamori, Masayuki, Yamamoto, Daisuke, Ishiyama, Naoko, Lee, Chang Nyoung, Choi, Mi Song, Jeong, Ha‐Neul, Lee, Hyung Seok, Hwang, Eun Bi, Visser, Marianne, Casasnovas, Carlos, Aguilo, Maria Antonia Alberti, Tejedor, Exuperio Diez, Rivera, Francisco Javier Rodriguez, Fernandez‐Fournier, Mireya, Sastre, Maria, Carbonell, Josep Gamez, Piehl, Fredrik, Hietala, Albert, Sengun, Ihsan, Orsel, Gulsah, Semiz, Cansu, Cavdar, Tugce Kirbas, Sohail, Faisal, Arndt, Heike, Salajegheh, Mohammed, Bravver, Elena, Plevka, Sarah, Guptill, Jeffrey, Hobson‐Webb, Lisa, Pascuzzi, Robert, Mosmiller, Betsy, Ventura, Nicholas, Carter, Cynthia, Sattar, Nadia, Daniels, Kevin, Jeffery, Dennis, Khoury, Julie, Dimitrova, Diana, Seelbach, Jenna, Paulose, Shirley, Bhavaraju‐Sanka, Ratna, Jones, Floyd, Roy, Gulmohor, Mathew, Veena, Echevarria, Laura, Shabbir, Yasmeen, Barohn, Richard, Glenn, Melanie, Johns, Kathryn, Naunton, Kerry, Bilsker, Martin, Karam, Chafic, Vu, Tuan, Gong, Hui, Karimi, Sanaz, Akhter, Salma, Strom, Susan, Bertorini, Tulio, Henderson, Kendrick, Wheeler, Lauren, Nye, Joan
Published in Muscle & nerve (01.12.2021)
Published in Muscle & nerve (01.12.2021)
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Journal Article
Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study
Suzuki, Hidekazu, Fujita, Kenji P., Rugiero, Marcelo, Bettini, Mariela, Garcia, Alejandra Dalila, Smetcoren, Charlotte, Van Daele, Sien, Verjans, Sarah, Melo, Ana Paula, Rocha, Rosana, Gonçalves Geraldo, Jordana, Otavio Maia Gonçalves, Luiz, Renata Cubas Volpe, Luciana, Amaral Andrade, Ana Carolina, Cavalcante Lino, Valeria, Carrara, Fernanda, Siddiqi, Zaeem, Phan, Cecile, Junkerova, Jana, Zapletalova, Olga, Wolfova, Marcela, Eriksson, Jaana, Matolcsi, Judit, Garibaldi, Matteo, Saccà, Francesco, Mantegazza, Renato, Bonanno, Silvia, Camera, Giorgia, Pasanisi, Maria, Matsuse, Dai, Nakamura, Yumiko, Futono, Kazumi, Morikawa, Miyuki, Miyawaki, Eriko, Motomura, Masakatsu, Maeda, Yasuko, Takada, Kazumi, Akiyama, Tetsuya, Fukuda, Yusuke, Sato, Miwako, Kawamura, Tomohiro, Kobayashi, Hisako, Tsuda, Emiko, Hayashi, Takashi, Murahara, Takashi, Suzuki, Shuichiro, Noshiro, Mayuko, Yasuda, Ikuko, Homedes-Pedret, Christian, Julia Palacios, Natalia, Diez Tejedor, Exuperio, Rodriguez de Rivera, Francisco Javier, Cortes Vicente, Elena, Querol Gutierrez, Luis Antonio, Bjarbo, Lena, Meherremova, Arzu, Balkan, Bengu, Ugur, Muzeyyen, Aydinlik, Seda, Cavus, Hakan, Kutlu, Ayse, Veske, Adife, Sokratous, Giannis, Shah, Amna, Arndt, Heike, Wolfe, Gil, Patrick, Kara, Forshew, Dallas, Bravver, Elena, Hobson-Webb, Lisa, Beck, Kate, Pascuzzi, Robert, Kincaid, John, Kelley, Andrea, Ho, Doreen, Scala, Stephanie, Herbert, Carol, Weiner, Elaine, Marburger, Tessa, Dimitrova, Diana, Seelback, Jenna, Alsharabati, Mohammad, Nozaki, Kenkichi, Minton, Brian, Rosenfeld, Jeffrey, Shah, Jaimin, Smith, Lisa, Jerath, Nivedita, Swenson, Andrea, Dimachkie, Mazen, Wang, Yunxia, Johns, Kathryn, Hafer-Macko, Charlene, Young, Valerie, Sheldon, Danielle, Katzin, Lara, Droker, Brian, Lin, Frank, Pokala, Krishna, Weiss, Michael
Published in Journal of the neurological sciences (15.12.2019)
Published in Journal of the neurological sciences (15.12.2019)
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Journal Article
Consistent improvement with eculizumab across muscle groups in myasthenia gravis
Mantegazza, Renato, Chaves, Marcelo, Vidal, Gonzalo, Mercelis, Rudy, Van Damme, Philip, Vandenbussche, Nicolas, Vynckier, Jan, Tilkin, Petra, Feder, David, Bezerra Rosa, Bruno, Paiva, Gabriel, Fernanda Butinhao, Cristiani, Paula Macagnan, Ana, Pinto, Graziela, Souza Bulle Oliveira, Acary, Annes, Marcelo, Cavalcante Lino, Valeria, Pinto, Wladimir, Assis, Natália, Fernandes, Patrícia, Narayan, Jeffrey, Blackmore, Derrick, Vohanka, Stanislav, Chmelikova, Magda, Toncrova, Stanislava, Kurkova, Barbora, Zapletalova, Olga, Pedersen, Jane, Erälinna, Juha‐Pekka, Harrison, Tuula, Rozsa, Csilla, Di Pasquale, Antonella, Garibaldi, Matteo, Gori, Maria, Marano, Enrico, Bonanno, Silvia, Camera, Giorgia, Pasanisi, Maria, Uzawa, Akiyuki, Kobayashi, Eri, Samukawa, Makoto, Suzuki, Hidekazu, Miyawaki, Eriko, Kobayashi, Hisako, Osakada, Kaori, Miyazaki, Teiichiro, Yoshimura, Shunsuke, Tsurumaru, Masako, Hisahara, Shin, Kawamata, Jun, Ishiyama, Yoko, Noshiro, Mayuko, Takeyama, Rumi, Lee, HyeLim, Lee, JiEun, Park, SangAe, Homedes‐Pedret, Christian, Diaz‐Manera, Jordi, Antonio Querol Gutiérrez, Luis, Lopez Ruiz, Pedro, Rodriguez de Rivera, Francisco Javier, Meherremova, Arzu, Pinar Acar, Nazire, Kazim Onar, Musa, Davies, Debbie, Wolfe, Gil, Sanjak, Mohammed, Massey, Janice, Kincaid, John, Chaudhry, Vinay, Vytopil, Michal, Jara, Jordan, Weiner, Elaine, McKinnon, Naya, McKenna, Kaitlyn, Kohlrus, Stephanie, Darnell, Taylor, Lokaitis, Barbara, Padilla, Patricia, Hopkins, Steve, Nozaki, Kenkichi, Mumfrey‐Thomas, Sandi, Woodall, Amy, Maqsood, Fatima, Minton, Brian, Garcia, Rebekah, Ross Berger, Alan, Shabbir, Yasmeen, Gutmann, Ludwig, Jenci, Nicole, Fondaw, Alex, Sharma, Khema, Steele, Julie, Hart, Adam, Waheed, Waqar, Weiss, Michael, Downing, Sharon, Arnold, Thomas, Hewlett, Jasmine, Dicapua, Daniel
Published in Annals of clinical and translational neurology (01.08.2020)
Published in Annals of clinical and translational neurology (01.08.2020)
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