487 results on '"Genge, Angela"'
Search Results
52. COURAGE-ALS: a randomized, double-blind phase 3 study designed to improve participant experience and increase the probability of success
- Author
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Shefner, Jeremy M., primary, Al-Chalabi, Ammar, additional, Andrews, Jinsy A., additional, Chio, Adriano, additional, De Carvalho, Mamede, additional, Cockroft, Bettina M., additional, Corcia, Philippe, additional, Couratier, Philippe, additional, Cudkowicz, Merit E., additional, Genge, Angela, additional, Hardiman, Orla, additional, Heiman-Patterson, Terry, additional, Henderson, Robert D., additional, Ingre, Caroline, additional, Jackson, Carlayne E., additional, Johnston, Wendy, additional, Lechtzin, Noah, additional, Ludolph, Albert, additional, Maragakis, Nicholas J., additional, Miller, Timothy M., additional, Mora Pardina, Jesus S., additional, Petri, Susanne, additional, Simmons, Zachary, additional, Van Den Berg, Leonard H., additional, Zinman, Lorne, additional, Kupfer, Stuart, additional, Malik, Fady I., additional, Meng, Lisa, additional, Simkins, Tyrell J., additional, Wei, Jenny, additional, Wolff, Andrew A., additional, and Rudnicki, Stacy A., additional
- Published
- 2023
- Full Text
- View/download PDF
53. Diagnostic delay in amyotrophic lateral sclerosis
- Author
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Gwathmey, Kelly G., primary, Corcia, Philippe, additional, McDermott, Chris J., additional, Genge, Angela, additional, Sennfält, Stefan, additional, de Carvalho, Mamede, additional, and Ingre, Caroline, additional
- Published
- 2023
- Full Text
- View/download PDF
54. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study
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Howard, James F, primary, Bresch, Saskia, additional, Genge, Angela, additional, Hewamadduma, Channa, additional, Hinton, John, additional, Hussain, Yessar, additional, Juntas-Morales, Raul, additional, Kaminski, Henry J, additional, Maniaol, Angelina, additional, Mantegazza, Renato, additional, Masuda, Masayuki, additional, Sivakumar, Kumaraswamy, additional, Śmiłowski, Marek, additional, Utsugisawa, Kimiaki, additional, Vu, Tuan, additional, Weiss, Michael D, additional, Zajda, Małgorzata, additional, Boroojerdi, Babak, additional, Brock, Melissa, additional, de la Borderie, Guillemette, additional, Duda, Petra W, additional, Lowcock, Romana, additional, Vanderkelen, Mark, additional, Leite, M Isabel, additional, Sembinelli, Dylan, additional, Teitelbaum, Jeanne, additional, Nicolle, Michael, additional, Bernard, Emilien, additional, Svahn, Juliette, additional, Spinazzi, Marco, additional, Stojkovic, Tanya, additional, Demeret, Sophie, additional, Weiss, Nicolas, additional, Le Guennec, Loïc, additional, Messai, Sihame, additional, Tranchant, Christine, additional, Nadaj-Pakleza, Aleksandra, additional, Chanson, Jean-Baptiste, additional, Suliman, Muhtadi, additional, Zaidi, Leila, additional, Tard, Celine, additional, Lecointe, Peggy, additional, Zschüntzsch, Jana, additional, Schmidt, Jens, additional, Glaubitz, Stefanie, additional, Zeng, Rachel, additional, Scholl, Matthias, additional, Kowarik, Markus, additional, Ziemann, Ulf, additional, Krumbholz, Markus, additional, Martin, Pascal, additional, Ruschil, Christoph, additional, Dünschede, Jutta, additional, Kemmner, Roswitha, additional, Rumpel, Natalie, additional, Berger, Benjamin, additional, Totzeck, Andreas, additional, Hagenacker, Tim, additional, Stolte, Benjamin, additional, Iorio, Raffaele, additional, Evoli, Amelia, additional, Falso, Silvia, additional, Antozzi, Carlo, additional, Frangiamore, Rita, additional, Vanoli, Fiammetta, additional, Rinaldi, Elena, additional, Deguchi, Kazushi, additional, Minami, Naoya, additional, Nagane, Yuriko, additional, Suzuki, Yasushi, additional, Ishida, Sayaka, additional, Suzuki, Shigeaki, additional, Nakahara, Jin, additional, Nagaoka, Astushi, additional, Yoshimura, Shunsuke, additional, Konno, Shingo, additional, Tsuya, Youko, additional, Uzawa, Akiyuki, additional, Kubota, Tomoya, additional, Takahashi, Masanori, additional, Okuno, Tatsusada, additional, Murai, Hiroyuki, additional, Gilhus, Nils Erik, additional, Boldingh, Marion, additional, Rønning, Tone Hakvåg, additional, Chyrchel-Paszkiewicz, Urszula, additional, Kumor, Klaudiusz, additional, Zielinski, Tomasz, additional, Banaszkiewicz, Krzysztof, additional, Błaż, Michał, additional, Kłósek, Agata, additional, Świderek-Matysiak, Mariola, additional, Szczudlik, Andrzej, additional, Paśko, Aneta, additional, Szczechowski, Lech, additional, Banach, Marta, additional, Ilkowski, Jan, additional, Kapetanovic Garcia, Solange, additional, Ortiz Bagan, Patricia, additional, Belén Cánovas Segura, Ana, additional, Turon Sans, Joana, additional, Vidal Fernandez, Nuria, additional, Cortes Vicente, Elena, additional, Rodrigo Armenteros, Patricia, additional, Ashraghi, Mohammad, additional, Cavey, Ana, additional, Haslam, Liam, additional, Emery, Anna, additional, Liow, Kore, additional, Yegiaian, Sharon, additional, Barboi, Alexandru, additional, Vazquez, Rosa Maria, additional, Lennon, Joshua, additional, Pascuzzi, Robert M, additional, Bodkin, Cynthia, additional, Guingrich, Sandra, additional, Comer, Adam, additional, Bromberg, Mark, additional, Janecki, Teresa, additional, Saba, Sami, additional, Tellez, Marco, additional, Elsheikh, Bakri, additional, Freimer, Miriam, additional, Heintzman, Sarah, additional, Govindarajan, Raghav, additional, Guptill, Jeffrey, additional, Massey, Janice M, additional, Juel, Vern, additional, Gonzalez, Natalia, additional, Habib, Ali A, additional, Mozaffar, Tahseen, additional, Korb, Manisha, additional, Goyal, Namita, additional, Machemehl, Hannah, additional, Manousakis, Georgios, additional, Allen, Jeffrey, additional, Harper, Emily, additional, Farmakidis, Constantine, additional, Saavedra, Lilli, additional, Dimachkie, Mazen, additional, Pasnoor, Mamatha, additional, Akhter, Salma, additional, Beydoun, Said, additional, McIlduff, Courtney, additional, Nye, Joan, additional, Roy, Bhaskar, additional, Munro Sheldon, Bailey, additional, Nowak, Richard, additional, Barnes, Benjamin, additional, Rivner, Michael, additional, Suresh, Niraja, additional, Shaw, Jessica, additional, Harvey, Brittany, additional, Lam, Lucy, additional, Thomas, Nikki, additional, Chopra, Manisha, additional, Traub, Rebecca E, additional, Jones, Sarah, additional, Wagoner, Mary, additional, Smajic, Sejla, additional, Aly, Radwa, additional, Katz, Jonathan, additional, Chen, Henry, additional, Miller, Robert G, additional, Jenkins, Liberty, additional, Khan, Shaida, additional, Khatri, Bhupendra, additional, Sershon, Lisa, additional, Pavlakis, Pantelis, additional, Holzberg, Shara, additional, Li, Yuebing, additional, Caristo, Irys B, additional, Marquardt, Robert, additional, Hastings, Debbie, additional, Rube, Jacob, additional, Lisak, Robert P, additional, Choudhury, Aparna, additional, Ruzhansky, Katherine, additional, Sachdev, Amit, additional, Shin, Susan, additional, Bratton, Joan, additional, Fetter, Mary, additional, McKinnon, Naya, additional, McKinnon, Jonathan, additional, Sissons-Ross, Laura, additional, Sahu, Amos, additional, and Distad, B Jane, additional
- Published
- 2023
- Full Text
- View/download PDF
55. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study
- Author
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Bril, Vera, primary, Drużdż, Artur, additional, Grosskreutz, Julian, additional, Habib, Ali A, additional, Mantegazza, Renato, additional, Sacconi, Sabrina, additional, Utsugisawa, Kimiaki, additional, Vissing, John, additional, Vu, Tuan, additional, Boehnlein, Marion, additional, Bozorg, Ali, additional, Gayfieva, Maryam, additional, Greve, Bernhard, additional, Woltering, Franz, additional, Kaminski, Henry J, additional, Genge, Angela, additional, Massie, Rami, additional, Berube, Maxime, additional, Bril, Vera, additional, Daniyal, Lubna, additional, Mannan, Shabber, additional, Ng, Eduardo, additional, Raman, Ritesh Rohan Raghu, additional, Sarpong, Evelyn, additional, Alcantara, Monica, additional, Dionne, Annie, additional, Siddiqi, Zaeem, additional, Blackmore, Derrick, additional, Hussain, Faraz, additional, Matte, Genevieve, additional, Botez, Stephan, additional, Tyblova, Michaela, additional, Jakubikova, Michala, additional, Junkerova, Jana, additional, Witting, Nanna, additional, Holm-Yildiz, Sonja, additional, Stemmerik, Mads, additional, Andersen, Henning, additional, Obál, Izabella, additional, Solé, Guilhem, additional, Mathis, Stéphane, additional, Violleau, Marie-Hélène, additional, Tranchant, Christine, additional, Messai, Sihame, additional, Chanson, Jean-Baptiste, additional, Nadaj-Pakleza, Aleksandra, additional, Verloes, Arnaud, additional, Zaidi, Leila, additional, Gambella, Manuela, additional, Cavalli, Michele, additional, Stojkovic, Tanya, additional, Demeret, Sophie, additional, Le Guennec, Loic, additional, Querin, Giorgia, additional, Weiss, Nicolas, additional, Masingue, Marion, additional, Magy, Laurent, additional, Ghorab, Karima, additional, Rukhadze, Ia, additional, Tsiskaridze, Alexander, additional, Janelidze, Marina, additional, Margania, Temur, additional, Then Bergh, Florian, additional, Hänsel, Eike, additional, Kalb, Andrea, additional, Meilick, Bianca, additional, Reuschel, Mandy, additional, Teußer, Lars-Malte, additional, Unterlauft, Astrid, additional, Goedel, Clemens, additional, Hagenacker, Tim, additional, Totzeck, Andreas, additional, Stolte, Benjamin, additional, Blaes, Franz, additional, Bindler, Christine, additional, Tsoutsikas, Vasilios, additional, Roediger, Annekathrin, additional, Geis, Christian, additional, Schmidt, Jens, additional, Zschüntzsch, Jana, additional, Schwarz, Margret, additional, Meyer, Stefanie, additional, Kummer, Karsten, additional, Glaubitz, Stefanie, additional, Zeng, Rachel, additional, Wiendl, Heinz, additional, Klotz, Luisa, additional, Lammerskitten, Anna, additional, Lünemann, Jan, additional, Diószeghy, Péter, additional, Maggi, Lorenzo, additional, Rinaldi, Elena, additional, Gastaldi, Matteo, additional, Mazzacane, Federico, additional, Businaro, Pietro, additional, Iorio, Raffaele, additional, Antonini, Giovanni, additional, Fionda, Laura, additional, Rinaldi, Rita, additional, Rossi, Simone, additional, Habetswallner, Francesco, additional, Tuccillo, Francesco, additional, Umehara, Haruna, additional, Uenaka, Eiko, additional, Takahashi, Masanori, additional, Higashi, Keiko, additional, Kinoshita, Makoto, additional, Yoneda, Emika, additional, Nakamura, Noriko, additional, Fujita, Saeka, additional, Kubota, Tomoya, additional, Ono, Masami, additional, Yamamoto, Sana, additional, Hatano, Taku, additional, Oikoshi, Kazuki, additional, Yokoyama, Kazumasa, additional, Oji, Yutaka, additional, Tomizawa, Yuji, additional, Uzawa, Akiyuki, additional, Yasuda, Manato, additional, Akita, Sachiko, additional, Ozawa, Yukiko, additional, Onishi, Yosuke, additional, Takaki, Miki, additional, Yamada, Hiromi, additional, Minemoto, Kanako, additional, Sanko, Miki, additional, Izawa, Nanae, additional, Nakayama, Mayumi, additional, Masuda, Masayuki, additional, Tsuji, Rune, additional, Ido, Nobuhiro, additional, Hyodo, Yumi, additional, Okubo, Yoshihiko, additional, Minohara, Akiko, additional, Haraguchi, Nana, additional, Naito, Makiko, additional, Yoshida, Seiko, additional, Fukushige, Yuri, additional, Tsujino, Akira, additional, Nagaoka, Atsushi, additional, Miyazaki, Teiichiro, additional, Yoshimura, Shunsuke, additional, Hirayama, Takuro, additional, Shima, Tomoaki, additional, Okamoto, Naoko, additional, Matsumoto, Riki, additional, Sekiguchi, Kenji, additional, Ueda, Takehiro, additional, Chihara, Norio, additional, Kirimura, Mari, additional, Sunagawa, Emi, additional, Suzuki, Ayaka, additional, Suzuki, Shigeaki, additional, Wada, Aozora, additional, Ishizuchi, Kei, additional, Suzuki, Yasushi, additional, Yata, Mitsuo, additional, Komatsu, Yuka, additional, Tsukita, Kenichi, additional, Watanabe, Genya, additional, Sato, Kazuki, additional, Kawasaki, Emiko, additional, Yamamoto, Naoki, additional, Ono, Hirohiko, additional, Tsuda, Tomoko, additional, Ohashi, Shigeki, additional, Fujisawa, Yuka, additional, Yokota, Yumiko, additional, Nagane, Yuriko, additional, Ayumi, Kameda, additional, Takematsu, Yuka, additional, Naito, Hiroyuki, additional, Kuwada, Kumiko, additional, Rejdak, Konrad, additional, Szklener, Sebastian, additional, Kitowska, Monika, additional, Derkacz, Kandyda, additional, Berkowicz, Tomasz, additional, Budzinska, Paulina, additional, Halas, Marek, additional, Zaslavskiy, Leonid, additional, Skornyakova, Evgeniya, additional, Kotov, Sergey, additional, Novikova, Ekaterina, additional, Sidorova, Olga, additional, Goldobin, Vitalii, additional, Alekseeva, Tatiana, additional, Isabekova, Patimat, additional, Malkova, Nadezhda, additional, Korobko, Denis, additional, Djordjevic, Gordana, additional, Stojanov, Aleksandar, additional, Peric, Stojan, additional, Lavrnic, Dragana, additional, Bozovic, Ivo, additional, Palibrk, Aleksa, additional, Casasnovas, Carlos, additional, Nedkova-Hristova, Velina, additional, Vidal Fernández, Nuria, additional, Cortés Vicente, Elena, additional, Querol Gutiérrez, Luis, additional, Salvadó Figueras, Maria, additional, Canovas Segura, Anna, additional, Juntas Morales, Raúl, additional, Sanchez Tejerina, Daniel, additional, Saiz, Albert, additional, Blanco Morgado, Yolanda, additional, Llufriú Durán, Sara, additional, Sepúlveda Gázquez, María, additional, Martínez Hernández, Eugenia María, additional, Gutiérrez Gutiérrez, Gerardo, additional, Iniesta, Paqui, additional, Meca Lallana, José, additional, Guo, Yuh-Cherng, additional, Chiu, Hou-Chang, additional, Yeh, Jiann-Horng, additional, Chen, Ya Hui, additional, Lee, Mei Fen, additional, Lee, Yi-Chung, additional, Lai, Kuan Lin, additional, Beydoun, Said, additional, Akhter, Salma, additional, Lam, Lucy, additional, Thomas, Alisha, additional, Rivner, Michael, additional, Quarles, Brandy, additional, Lange, Dale, additional, Holzberg, Shara, additional, Pavlakis, Pantelis, additional, Goutham, Ashwathy, additional, Kaminski, Henry, additional, Aly, Radwa, additional, Ashworth, Lisa, additional, Bender, Kathryn, additional, Bond, Karie, additional, Buckner, Joanne, additional, Byerly, Sara, additional, Caress, James, additional, Clemons, Jessyca, additional, Farmer, Asha, additional, Franklin, Catherine, additional, Harris, Summer, additional, Hiatt, Meredith, additional, Gandhi Mehta, Rachana, additional, Miller, Gina, additional, Smith, Lynn, additional, Smith, Rose, additional, Strittmatter, Brian, additional, Mozaffar, Tahseen, additional, Hernandez, Isela, additional, Moulton, Kelsey, additional, Karam, Chafic, additional, Ravikumar, Pranali, additional, Lomen-Hoerth, Catherine, additional, Rosow, Laura, additional, George, Hannah, additional, Irodenko, Viktoriya, additional, Denny, Carol, additional, Hanson, Bart, additional, Klein, Sara, additional, Martinez-Thompson, Jennifer, additional, Naddaf, Elie, additional, Padgett, Denny, additional, Sorenson, Eric, additional, L Sultze, Jane, additional, Weis, Delena, additional, Rezania, Kourosh, additional, Thonhoff, Jason, additional, Shroff, Sheetal, additional, Pascuzzi, Robert, additional, Micheels, Angela, additional, Bodkin, Cynthia, additional, Comer, Adam, additional, Baras, Gelasio, additional, Wagner, Renee, additional, Mahuwala, Zabeen, additional, Ryan, Stephen, additional, Su, Kai, additional, Sharma, Khema, additional, Brown, Andrew, additional, and Liow, Kore, additional
- Published
- 2023
- Full Text
- View/download PDF
56. Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
- Author
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Miller, Timothy M, Cudkowicz, Merit E, Andrews, Jinsy A, Hesters, Adele, Kermorvant, Hugo, Lacomblez, Lucette, Forestier, Nadine Le, Lenglet, Thimotée, Retail, Maryvonne, Ruiz Del Mar Amador, Maria, Salachas, François, Shotar, Eimad, Sourour, Nader, Babu, Suma, Dorst, Johannes, Froehlich, Elke, Fromm, Andrea, Kandler, Katharina, Langer, Eva, Leichtle, Sarah, Ludolph, Albert, Mayer, Kristina, Michels, Sebastian, Raubold, Sabine, Benatar, Michael, Schuster, Joachim, Weiland, Ulrike, Wiesenfarth, Maximilian, Witzel, Simon, Calvo, Andrea, Canosa, Antonio, Casale, Federico, Chiò, Adriano, Fuda, Giuseppe, Grassano, Maurizio, McDermott, Christopher J, Marchese, Giulia, Moglia, Cristina, Palumbo, Francesca, Salamone, Paolina, Ajiki, Takahiro, Akasaka, Aya, Ando, Masahiro, Arata, Hitoshi, Asuka, Kitamura, Baba, Kosuke, Cochrane, Thos, Bekku, Goichi, Chiba, Tomoya, Date, Yugaku, Eriko, Takeuchi, Hashiguchi, Akihiro, Hatatori, Ritsuko, Hayano, Eri, Hayashi, Yuto, Higashi, Keiko, Higuchi, Eriko, Chary, Sowmya, Hiramatsu, Yu, Horikawa, Rui, Ikenaka, Kensuke, Ishiura, Hiroyuki, Ito, Daisuke, Kawai, Sachiko, Kikuchi, Junko, Kuzuyama, Haruko, Li, Xuehong, Matsumoto, Chika, Chew, Sheena, Matsuura, Eiji, Michizono, Kumiko, Mitsui, Jun, Mitsutake, Akihiko, Mochizuki, Hideki, Nagamatsu, Akemi, Nagano, Seiichi, Nakamura, Tomonori, Naruse, Hiroya, Ogasawara, Asuka, Zhu, Han, Okada, Kensuke, Okamoto, Yuji, Okuno, Tatsusada, Oyama, Satoshi, Ozono, Tatsuhiko, Sakiyama, Yusuke, Sakuishi, Kaori, Seki, Morinobu, Shibata, Shota, Shimizu, Mikito, Wu, Fan, Takahata, Katsunori, Takahito, Yoshizaki, Takashima, Hiroshi, Takeichi, Hiroko, Tashiro, Yuichi, Toda, Tatsushi, Tomizu, Yuki, Tomoya, Wadayama, Ujiakira, Nishiike, Yashita, Daiki, Nestorov, Ivan, Al-Chalabi, Ammar, Alix, James, Bangalore, Priyadarshini, Blackburn, Daniel, Chiwera, Theresa, Clegg, Rosie, Collins, Alexis, Cooper-Knock, Jonathan, Emery, Anna, Franklin, John, Genge, Angela, Graham, Danielle, Green, Louisa, Harvey, Callum, Hobson, Esther, Islam, Mahjabim, Jenkins, Thomas Michael, Kazoka, Mbombe, Kelly, Gillian, Korley, Mercy, Madarshahaian, Daniel, Mayl, Keith, Sun, Peng, McDermott, Christopher John, Radford, Alex, Shaw, Christopher, Shaw, Pamela J, Sidebottom, Joe, Smart, Lynne, Sreedharan, Jemeen, Stone, Ben, Tsironis, Theocharis, Tuddenham, Lee, McNeill, Manjit, Verber, Nick, Wollff, Helen, Young, Stacy, Zis, Panagiotis, Adamo, Ashley, Ahmed, Arubah, Ajroud-Driss, Senda, Alameda, Gustave, Arcila-Londono, Ximena, Fanning, Laura, Baird, Candy, Bazan, Tracy, Berry, James, Bordeau, Jane, Bradford, Wendy, Brook, Nyda, Brown, Lauren, Bucelli, Robert C, Ferguson, Toby A, Buckner, Katherine, Budler, Michael W, Burba, Lindita, Burke, Katherine, Calhoun, Ashley D, Campbell, Sarah, Carey, Judith, Caristo, Irys B, Carty, Simon, Chan, Emmanuel, Fradette, Stephanie, Chaudhry, Vinay, Chen, Ricky, Chow, Saephanh, Clawson, Lora L, Clemens, Mitchell, Cloninger, Suzann E, Coleman-Wood, Krista, Cooper, Thomas N, Cummings, Arlena, Daniels, Jacquelyn, VALOR, DeSaro, Pamela, DeWitt, Michelle, Dedi, Brixhilda, Dempsey, Debbie, Denny, Carol, Doherty, Jenna, Doherty, Leana, Donahue, Megan, Doyle, Michael, Duncan, Jessie, Group, OLE Working, Elman, Lauren, Eloge, Christine M, Echiti, Desirae R, Ferrey, Dominic, Fournier, Christina, Fukumura, Yuriko, Gallagher, Katherine, Garaycoa, Jessica, Garrett, Mark, Gibson, Richard L, Beullens, Lien, Gifford, Ryan, Glass, Jonathan D, Gogol, Danuta, Golden, Shea, Gonzalez, Alexa, Goodman, Ira, Goolsby, Christopher, Goslin, Kimberly, Goulbourne, Michael, Granit, Volkan, Claeys, Kristl, Grignon, Anne-Laure, GuhaRay, Adreeja, Guide, Debra, Gundogdu, Melek Betul, Gutierrez, Gil, Hastings, Debbie, Hayzen, Colleen, Herzog, Hilary, Holloway, Raegan, Jacobs, Gabriel, Claeys, Thomas, Jacobsen, Bill, James, Virginia, Jenkins, Liberty, Jockel-Balsarotti, Jennifer, Johnson, Linda Carol, Jose, Sunil, Joslin, Benjamin, Karanja, Elizabeth, Katz, Jonathan, Keener, Anthony, Couwelier, Goedele, Kittle, Gale, Klein, Sara, Kreple, Collin, Rebecca, Rebecca, Kuenzler, Kuenzler, Kusnir, Jorge, Labbe, Kristen, Lachica-Encinas, Nicolet, Ladha, Shafeeq, Leimer, Lesli, D'Hondt, Ann, Levy, Michael, Levy, Wendy, Li, Yingji, Likanje, Marie-France, Livigni, Rebecca, Locatelli, Eduardo, Luppino, Sarah, Malcolm, Amber, Maragakis, Nicholas, Marin, Horia, Debien, Elisa, Markowitz, Clyde, Markway, Jesse, McCaffrey, Alexandra, McCoy, Arita, McCoy Gross, Kelly, Mehta, Kush, Meyer, Robert, Milan, Jennifer, Miller, Timothy, Miller, Robert G, de Keersmaecker, Sebastiaan, Morales, Francisco, Mosmiller, Elizabeth, Mott, Donovan, Moulton, Kelsey, Murphy, Christine A, Negron, Tirso, Nelson, Cassandra, Newman, Daniel S, Nissinen, Janne Kristoffer, Norman, Andrew, Della Faille, Laetitia, Ohkubo, Takuya, Olney, Nicholas, Ortiz, Natasha, Oskarsson, Bjorn, Pace, Mitchell, Packard, Kathleen, Padgett, Denny, Paganoni, Sabrina, Paredes, Maria E, Parker, Elizabeth, Delmotte, Koen, Partlow, Ann, Pattee, Gary L, Paulett, Jany, Pelot, Antoinette, Pfeifer, Kyle M, Pijanowski, Olivia, Pioro, Erik, Polak, Meraida, Prakash, Ahalya, Previte, Rosemarie, Depoortere, Sofie, Pukenas, Bryan, Quinn, Colin, Ravits, John, Razavi, Ryan, Regan, Tyler, Riley, Kristen M, Roth, Heather, Sanders, Danica, Scalia, Jennifer, Schmidt, Emma, de Velder, Laura, Schwen, Edward, Shah, Jaimin, Shah, Stuti, Shefner, Jeremy, Sheldon, Danielle, Simmons, Karon, Singh, Navneet K, Singleton, Jessica, Smiley, Richard, Smith, William B, Dobbels, Laurens, Smith, Sean, Sotirchos, Elias, Sorenson, Eric, Staff, Nathan, Steele, Julie, Steijlen, Kara, Stirrat, Taylor, Stoica, George S, Strong, Stephanie, Sufit, Robert, Sobue, Gen, Gijs, Jeroen, Sultze, Jane, Swartz, Amy, Szymanski, April, Tay, Anna, Thakore, Nimish, Thiessen, Diana, Thotala, Sukrutha, Trudell, Randall G, Turcotte, Nicole, Turner, Michelle, Horckmans, Simon, Uchil, Alpa, Upadhyay, Vihar, Usman, Uzma, Vallis, Anne, Vaporean-Bussey, Danielle, Vladimirova, Valentine, Weber, Harli, Winbigler, Jennifer, Wojanowski, Heather, Wulf, Charlie, Lamaire, Nikita, Yasek, Julia, Yoo, Stephanie, Zivalic, Hannah, Cole, Alexandra, File, Greta, Foate, Jeremy, Mason, Deborah, Newton, Susan, Roberts, Stephen, Sellwood, Cory Dean, Liessens, Hannelore, Swan, James, Werno, Anja, Zhong, Cathy, Masrori, Pegah, Nysten, Celine, Schotte, Caroline, Serrien, Anouk, Swinnen, Bart, Tilkin, Petra, van Daele, Sien, Van Damme, Philip, Vynckier, Jan, Wouters, Anke, Abrahao, Agessandro, Angle, Mark, Badawy, Mohamed, Berube, Maxime, Bertone, Vanessa, Cooper, Sarah Marie, Dobrowolski, Peter, Fong, Helen, Hannouche, Matthew, Hartley, Denise, Hogan, Michael, Johnston, Wendy, Khalfallah, Yousra, Korngut, Lawrence, Kroetsch, Gina, Letourneau, Justin, Magnussen, Claire, Martinez, Jose, Massie, Rami, Mobach, Theodore, Mookshah, Jahan, Ozelsel, Timur, Parks, Andrea, Petrillo, Janet, Pfeffer, Gerald, Ludolph, Albert C, Pham, Shirley, Phung, Liane, Shiungsun, Rodney, Pi-Shan, Li, Santos, Denizart, Salmon, Kristiana, Saunders, Natalie, Sembinelli, Dylan, Tymkow, Kelsey, Wong, Berchman, Zinman, Lorne, Karlsborg, Merete, Pedersen Lomholt, Therese, Nilsson, Sigrid, Salvesen, Lisette, Skov, Pernille, Svenstrup, Kristen, Bruneteau, Gaelle, Calerencon, Frederic, and Guimaraes Costa, Raquel
- Subjects
Adult ,drug effects [Recovery of Function] ,Spinal ,Oligonucleotides ,blood [Neurofilament Proteins] ,administration & dosage [Oligonucleotides, Antisense] ,tofersen ,Injections ,blood [Amyotrophic Lateral Sclerosis] ,pharmacology [Oligonucleotides, Antisense] ,Superoxide Dismutase-1 ,Double-Blind Method ,Neurofilament Proteins ,Humans ,ddc:610 ,Antisense ,Injections, Spinal ,Biomarkers ,Recovery of Function ,Amyotrophic Lateral Sclerosis ,Oligonucleotides, Antisense ,blood [Biomarkers] ,drug therapy [Amyotrophic Lateral Sclerosis] ,therapeutic use [Oligonucleotides, Antisense] ,SOD1 protein, human ,General Medicine ,genetics [Superoxide Dismutase-1] ,genetics [Amyotrophic Lateral Sclerosis] ,cerebrospinal fluid [Biomarkers] ,cerebrospinal fluid [Superoxide Dismutase-1] ,cerebrospinal fluid [Amyotrophic Lateral Sclerosis] - Abstract
The intrathecally administered antisense oligonucleotide tofersen reduces synthesis of the superoxide dismutase 1 (SOD1) protein and is being studied in patients with amyotrophic lateral sclerosis (ALS) associated with mutations in SOD1 (SOD1 ALS).In this phase 3 trial, we randomly assigned adults with SOD1 ALS in a 2:1 ratio to receive eight doses of tofersen (100 mg) or placebo over a period of 24 weeks. The primary end point was the change from baseline to week 28 in the total score on the ALS Functional Rating Scale-Revised (ALSFRS-R; range, 0 to 48, with higher scores indicating better function) among participants predicted to have faster-progressing disease. Secondary end points included changes in the total concentration of SOD1 protein in cerebrospinal fluid (CSF), in the concentration of neurofilament light chains in plasma, in slow vital capacity, and in handheld dynamometry in 16 muscles. A combined analysis of the randomized component of the trial and its open-label extension at 52 weeks compared the results in participants who started tofersen at trial entry (early-start cohort) with those in participants who switched from placebo to the drug at week 28 (delayed-start cohort).A total of 72 participants received tofersen (39 predicted to have faster progression), and 36 received placebo (21 predicted to have faster progression). Tofersen led to greater reductions in concentrations of SOD1 in CSF and of neurofilament light chains in plasma than placebo. In the faster-progression subgroup (primary analysis), the change to week 28 in the ALSFRS-R score was -6.98 with tofersen and -8.14 with placebo (difference, 1.2 points; 95% confidence interval [CI], -3.2 to 5.5; P = 0.97). Results for secondary clinical end points did not differ significantly between the two groups. A total of 95 participants (88%) entered the open-label extension. At 52 weeks, the change in the ALSFRS-R score was -6.0 in the early-start cohort and -9.5 in the delayed-start cohort (difference, 3.5 points; 95% CI, 0.4 to 6.7); non-multiplicity-adjusted differences favoring early-start tofersen were seen for other end points. Lumbar puncture-related adverse events were common. Neurologic serious adverse events occurred in 7% of tofersen recipients.In persons with SOD1 ALS, tofersen reduced concentrations of SOD1 in CSF and of neurofilament light chains in plasma over 28 weeks but did not improve clinical end points and was associated with adverse events. The potential effects of earlier as compared with delayed initiation of tofersen are being further evaluated in the extension phase. (Funded by Biogen; VALOR and OLE ClinicalTrials.gov numbers, NCT02623699 and NCT03070119; EudraCT numbers, 2015-004098-33 and 2016-003225-41.).
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- 2022
57. Subcutaneous batoclimab in generalized myasthenia gravis: Results from a Phase 2a trial with an open‐label extension.
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Nowak, Richard J., Breiner, Ari, Bril, Vera, Allen, Jeffrey A., Khan, Shaida, Levine, Todd, Jacobs, Daniel H., Sahagian, Gregory, Siddiqi, Zaeem A., Xu, Jing, Macias, William L., Benatar, Michael, Adams, Laurence, Genge, Angela, Habib, Ali, Hinton, John L., Holmlund, Tomas H., Lange, Dale J., Nicolle, Michael W., and Phan, Han C.
- Subjects
MYASTHENIA gravis ,IMMUNOGLOBULIN G ,RECEPTOR antibodies ,FC receptors ,SUBCUTANEOUS injections - Abstract
Objectives: To assess the safety, tolerability, and key pharmacodynamic effects of subcutaneous batoclimab, a fully human anti‐neonatal Fc receptor monoclonal antibody, in patients with generalized myasthenia gravis and anti‐acetylcholine receptor antibodies. Methods: A Phase 2a, proof‐of‐concept, randomized, double‐blind, placebo‐controlled trial is described. Eligible patients were randomized (1:1:1) to receive once‐weekly subcutaneous injections of batoclimab 340 mg, batoclimab 680 mg, or matching placebo for 6 weeks. Subsequently, all patients could enter an open‐label extension study where they received batoclimab 340 mg once every 2 weeks for 6 weeks. Primary endpoints were safety, tolerability, and change from baseline in total immunoglobulin G, immunoglobulin G subclasses, and anti‐acetylcholine receptor antibodies at 6 weeks post‐baseline. Secondary endpoints included changes from baseline to 6 weeks post‐baseline for Myasthenia Gravis Activities of Daily Living, Quantitative Myasthenia Gravis, Myasthenia Gravis Composite, and revised 15‐item Myasthenia Gravis Quality of Life scores. Results: Seventeen patients were randomized to batoclimab 680 mg (n = 6), batoclimab 340 mg (n = 5), or placebo (n = 6). Batoclimab was associated with significantly greater reductions in total immunoglobulin G and anti‐acetylcholine receptor antibodies from baseline to 6 weeks post‐baseline than placebo. Reductions in immunoglobulin G subclasses were generally consistent with total immunoglobulin G. While clinical measures showed directionally favorable improvements over time, the study was not powered to draw conclusions about therapeutic efficacy. No safety issues were identified. Interpretation: The safety profile, pharmacodynamics, and preliminary clinical benefits observed in this study support further investigation of subcutaneous batoclimab injections as a potential patient‐administered therapy for seropositive generalized myasthenia gravis. [ABSTRACT FROM AUTHOR]
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- 2024
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58. A prospective harmonized multicenter DTI study of cerebral white matter degeneration in ALS
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Kalra, Sanjay, Müller, Hans-Peter, Ishaque, Abdullah, Zinman, Lorne, Korngut, Lawrence, Genge, Angela, Beaulieu, Christian, Frayne, Richard, Graham, Simon J., and Kassubek, Jan
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- 2020
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59. RAISE-XT: An interim analysis of safety and efficacy in an open-label extension study of zilucoplan in patients with myasthenia gravis (P1-5.007)
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Freimer, Miriam, primary, Leite, M. Isabel, additional, Genge, Angela, additional, Hussain, Yessar, additional, Kaminski, Henry J., additional, Mantegazza, Renato, additional, Utsugisawa, Kimiaki, additional, Vu, Tuan, additional, Duda, Petra W., additional, Boroojerdi, Babak, additional, Vanderkelen, Mark, additional, Lowcock, Romana, additional, and Howard, James F., additional
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- 2023
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60. Phase 3, Open-Label, Multicenter Safety Study of Oral Edaravone in Patients With Amyotrophic Lateral Sclerosis (MT-1186-A01): 48-Week Results (P6-4.001)
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Genge, Angela, primary, Pattee, Gary, additional, Sobue, Gen, additional, Couratier, Philippe, additional, Selness, Daniel, additional, Bidani, Sachin, additional, Hirai, Manabu, additional, Sakata, Takeshi, additional, Salah, Alejandro, additional, and Apple, Stephen, additional
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- 2023
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61. Spinraza in Adults with Spinal Muscular Atrophy (SAS) – 14 month results (S33.010)
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Zaidman, Craig, primary, Proud, Crystal, additional, Thonhoff, Jason, additional, Rad, Nassim, additional, Ho, Doreen, additional, Chu, MaryLynn, additional, Ladha, Shafeeq, additional, Crawford, Thomas, additional, Nayar, Shakti, additional, Genge, Angela, additional, Frey, Margaret, additional, Heatwole, Chad, additional, and Lew, Daphne, additional
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- 2023
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62. Motor Outcomes to Validate Evaluations in Facioscapulohumeral muscular dystrophy (MOVE FSHD): Preliminary Baseline Characteristics (S7.004)
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Walker, Michaela, primary, Butterfield, Russell, additional, Day, John, additional, Eichinger, Katy, additional, Elsheikh, Bakri, additional, Friedman, Seth, additional, Genge, Angela, additional, Higgs, Kiley, additional, Johnson, Nicholas, additional, Jones, Peter, additional, Leung, Doris, additional, Lewis, Leann, additional, Lochmuller, Hanns, additional, O'Ferrall, Erin, additional, Martens, William, additional, Shaw, Dennis, additional, Shieh, Perry, additional, Subramony, S, additional, Trivedi, Jaya, additional, Wang, Leo, additional, Wicklund, Matthew, additional, Tawil, Alrabi, additional, and Statland, Jeffrey, additional
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- 2023
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63. Health utilities and quality-adjusted life years for patients with amyotrophic lateral sclerosis receiving reldesemtiv or placebo in FORTITUDE-ALS
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Gebrehiwet, Paulos, primary, Meng, Lisa, additional, Rudnicki, Stacy A., additional, Sarocco, Phil, additional, Wei, Jenny, additional, Wolff, Andrew A., additional, Butzner, Michael, additional, Chiò, Adriano, additional, Andrews, Jinsy A., additional, Genge, Angela, additional, Hughes, Dyfrig A., additional, Jackson, Carlayne E., additional, Lechtzin, Noah, additional, Miller, Timothy M., additional, and Shefner, Jeremy M., additional
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- 2023
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64. Distinct Plasma Immune Profile in ALS Implicates sTNFR-II in pAMPK/Leptin Homeostasis
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Picher-Martel, Vincent, primary, Boutej, Hejer, additional, Vézina, Alexandre, additional, Cordeau, Pierre, additional, Kaneb, Hannah, additional, Julien, Jean-Pierre, additional, Genge, Angela, additional, Dupré, Nicolas, additional, and Kriz, Jasna, additional
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- 2023
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65. Efficacy and Safety of Ravulizumab, a Complement C5 Inhibitor, in Adults With Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial
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Neurologen, Projectafdeling ALS, Brain, Regenerative Medicine and Stem Cells, Genge, Angela, van den Berg, Leonard H, Frick, Glen, Han, Steve, Abikoff, Cori, Simmons, Adam, Lin, Qun, Patra, Kaushik, Kupperman, Erik, Berry, James D, Neurologen, Projectafdeling ALS, Brain, Regenerative Medicine and Stem Cells, Genge, Angela, van den Berg, Leonard H, Frick, Glen, Han, Steve, Abikoff, Cori, Simmons, Adam, Lin, Qun, Patra, Kaushik, Kupperman, Erik, and Berry, James D
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- 2023
66. Courage-als: a randomized, double-blind phase 3 study designed to improve participant experience and increase the probability of success
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Neurologen, Projectafdeling ALS, Brain, Regenerative Medicine and Stem Cells, Shefner, Jeremy M, Al-Chalabi, Ammar, Andrews, Jinsy A, Chio, Adriano, De Carvalho, Mamede, Cockroft, Bettina M, Corcia, Philippe, Couratier, Philippe, Cudkowicz, Merit E, Genge, Angela, Hardiman, Orla, Heiman-Patterson, Terry, Henderson, Robert D, Ingre, Caroline, Jackson, Carlayne E, Johnston, Wendy, Lechtzin, Noah, Ludolph, Albert, Maragakis, Nicholas J, Miller, Timothy M, Mora Pardina, Jesus S, Petri, Susanne, Simmons, Zachary, Van Den Berg, Leonard H, Zinman, Lorne, Kupfer, Stuart, Malik, Fady I, Meng, Lisa, Simkins, Tyrell J, Wei, Jenny, Wolff, Andrew A, Rudnicki, Stacy A, Neurologen, Projectafdeling ALS, Brain, Regenerative Medicine and Stem Cells, Shefner, Jeremy M, Al-Chalabi, Ammar, Andrews, Jinsy A, Chio, Adriano, De Carvalho, Mamede, Cockroft, Bettina M, Corcia, Philippe, Couratier, Philippe, Cudkowicz, Merit E, Genge, Angela, Hardiman, Orla, Heiman-Patterson, Terry, Henderson, Robert D, Ingre, Caroline, Jackson, Carlayne E, Johnston, Wendy, Lechtzin, Noah, Ludolph, Albert, Maragakis, Nicholas J, Miller, Timothy M, Mora Pardina, Jesus S, Petri, Susanne, Simmons, Zachary, Van Den Berg, Leonard H, Zinman, Lorne, Kupfer, Stuart, Malik, Fady I, Meng, Lisa, Simkins, Tyrell J, Wei, Jenny, Wolff, Andrew A, and Rudnicki, Stacy A
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- 2023
67. Cerebral degeneration in amyotrophic lateral sclerosis: A prospective multicenter magnetic resonance spectroscopy study
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Srivastava, Ojas, Hanstock, Chris, Chenji, Sneha, Mah, Dennell, Eurich, Dean, Ta, Daniel, Seres, Peter, Luk, Collin, Zinman, Lorne, Abrahao, Agessandro, Graham, Simon J., Genge, Angela, Korngut, Lawrence, Frayne, Richard, and Kalra, Sanjay
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- 2019
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68. Feasibility of Lung Volume Recruitment in Early Neuromuscular Weakness: A Comparison Between Amyotrophic Lateral Sclerosis, Myotonic Dystrophy, and Postpolio Syndrome
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Kaminska, Marta, Browman, Franceen, Trojan, Daria A., Genge, Angela, Benedetti, Andrea, and Petrof, Basil J.
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- 2015
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69. Oral edaravone demonstrated a favorable safety profile in patients with amyotrophic lateral sclerosis after 48 weeks of treatment
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Genge, Angela, primary, Pattee, Gary L., additional, Sobue, Gen, additional, Aoki, Masashi, additional, Yoshino, Hiide, additional, Couratier, Philippe, additional, Lunetta, Christian, additional, Petri, Susanne, additional, Selness, Daniel, additional, Bidani, Sachin, additional, Hirai, Manabu, additional, Sakata, Takeshi, additional, Salah, Alejandro, additional, Apple, Stephen, additional, Wamil, Art, additional, Kalin, Alexander, additional, and Jackson, Carlayne E., additional
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- 2022
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70. The future of ALS diagnosis and staging: where do we go from here?
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Genge, Angela, primary and Chio, Adriano, additional
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- 2022
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71. MiToS and King’s staging as clinical outcome measures in ALS: a retrospective analysis of the FORTITUDE-ALS trial
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Gebrehiwet, Paulos, primary, Meng, Lisa, additional, Rudnicki, Stacy A., additional, Sarocco, Phil, additional, Wei, Jenny, additional, Wolff, Andrew A., additional, Chiò, Adriano, additional, Andrews, Jinsy A., additional, Genge, Angela, additional, Jackson, Carlayne E., additional, Lechtzin, Noah, additional, Miller, Timothy M., additional, and Shefner, Jeremy M., additional
- Published
- 2022
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72. Motor cortex functional connectivity is associated with underlying neurochemistry in ALS
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Dey, Avyarthana, primary, Luk, Collin C, additional, Ishaque, Abdullah, additional, Ta, Daniel, additional, Srivastava, Ojas, additional, Krebs, Dennell, additional, Seres, Peter, additional, Hanstock, Chris, additional, Beaulieu, Christian, additional, Korngut, Lawrence, additional, Frayne, Richard, additional, Zinman, Lorne, additional, Graham, Simon, additional, Genge, Angela, additional, Briemberg, Hannah, additional, and Kalra, Sanjay, additional
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- 2022
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73. Rare and common variant analyses of amyotrophic lateral sclerosis in the French-Canadian genome
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Ross, Jay P., primary, Akçimen, Fulya, additional, Liao, Calwing, additional, Kwan, Karina, additional, Phillips, Daniel E., additional, Schmilovich, Zoe, additional, Spiegelman, Dan, additional, Genge, Angela, additional, Dupré, Nicolas, additional, Dion, Patrick A., additional, Farhan, Sali M.K., additional, and Rouleau, Guy A., additional
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- 2022
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74. Tofersen in adults with SOD1-ALS: phase 3 VALOR trial and open-label extension results
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Shaw, Pamela, primary, Miller, Timothy, additional, Cudkowicz, Merit, additional, Genge, Angela, additional, Sobue, Gen, additional, Nestorov, Ivan, additional, Graham, Dan- ielle, additional, Fanning, Laura, additional, Fradette, Stephanie, additional, and McNeill, Manjit, additional
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- 2022
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75. Clinical studies in amyotrophic lateral sclerosis
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Dorst, Johannes, primary and Genge, Angela, additional
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- 2022
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76. The future of ALS diagnosis and staging: where do we go from here?
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Genge, Angela and Chio, Adriano
- Subjects
- *
CAREGIVERS , *AMYOTROPHIC lateral sclerosis , *DELAYED diagnosis , *DIAGNOSIS , *EVIDENCE-based medicine - Abstract
Amyotrophic lateral sclerosis (ALS) is a rare, progressive multi-system neurodegenerative disorder. Its clinical presentation varies considerably leading to delays in diagnosis, which has dire consequences in a disease where early intervention is key to optimize outcomes and limit care giver burden. There are a range of diagnostic criteria available to aid ALS diagnosis, as well staging methods to assess disease progression. However, they all suffer from inter-rater variability, complexity, and confusion in use. Such difficulties, when medical appointment times are limited and becoming more virtually based, have the potential to amplify uncertainty and errors in ALS diagnosis and prognosis. This review provides a clinical overview of the best way to balance the needs of evidence-based medicine and the patient. We focus on ALS diagnostic criteria and staging systems currently in use in clinical practice and explore factors that could enhance diagnostic efficiency and assessment of disease progression. [ABSTRACT FROM AUTHOR]
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- 2023
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77. MiToS and King's staging as clinical outcome measures in ALS: a retrospective analysis of the FORTITUDE-ALS trial.
- Author
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Gebrehiwet, Paulos, Meng, Lisa, Rudnicki, Stacy A., Sarocco, Phil, Wei, Jenny, Wolff, Andrew A., Chiò, Adriano, Andrews, Jinsy A., Genge, Angela, Jackson, Carlayne E., Lechtzin, Noah, Miller, Timothy M., and Shefner, Jeremy M.
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AMYOTROPHIC lateral sclerosis ,RETROSPECTIVE studies - Abstract
To evaluate the Milano-Torino staging (MiToS) and King's staging systems as potential outcome measures for clinical trials in amyotrophic lateral sclerosis (ALS) by assessing these outcomes in FORTITUDE-ALS. This was a post hoc analysis of the phase 2b FORTITUDE-ALS trial (NCT03160898), a double-blind, randomized, dose-ranging, placebo-controlled, parallel-group study of reldesemtiv in patients with ALS. The treatment period was 12 weeks, with a follow-up assessment at week 16. Patients were retrospectively classified into MiToS and King's stages. Outcomes were the mean time maintaining baseline stage and risk of progression from the baseline stage to a later stage. The full analysis set consisted of 456 patients randomized 3:1 (reldesemtiv n = 342, placebo n = 114) who received at least one dose of double-blind study drug and had at least one post-baseline assessment. At baseline, MiToS and King's stages were balanced between the reldesemtiv and placebo groups: >99% of patients were in MiToS stage 0 or 1 and King's stage 1, 2 or 3. Time of maintaining the baseline stage was similar in both groups, for each staging system. The two staging systems exhibited considerably disparate results for risk of progression from baseline to a later stage: hazard ratio (HR) = 0.62 (95% confidence interval [CI] 0.38, 0.99) for MiToS and HR = 0.96 (95% CI 0.63, 1.44) for King's. This exploratory analysis showed the feasibility of MiToS and King's staging as potential outcome measures in ALS. Additional studies of these staging systems are needed to further explore their utility in ALS clinical trials. [ABSTRACT FROM AUTHOR]
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- 2023
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78. 24-Week Results From Phase 3 Study MT-1186-A01 an Open-Label, Multicenter Safety Study of Oral Edaravone in Subjects With Amyotrophic Lateral Sclerosis (P1-13.001)
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Genge, Angela, primary, Pattee, Gary L., additional, Sobue, Gen, additional, Couratier, Philippe, additional, Selness, Daniel, additional, Hirai, Manabu, additional, Sakata, Takeshi, additional, Salah, Alejandro, additional, and Apple, Stephen, additional
- Published
- 2022
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79. A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week Study of the Efficacy and Safety of Losmapimod in Subjects with FSHD: ReDUX4 (S23.007)
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Tawil, Rabi, primary, Statland, Jeffrey, additional, Wang, Leo, additional, Genge, Angela, additional, Sacconi, Sabrina, additional, Lochmuller, Hanns, additional, Leiva, David Reyes, additional, Manera, Jordi Diaz, additional, Alonso-Perez, Jorge, additional, Gomez, Nuria Muelas, additional, Padilla, Juan Vilchez, additional, Pestronk, Alan, additional, Gibson, Summer, additional, Goyal, Namita, additional, Hamel, Johanna, additional, Hayward, Lawrence, additional, Johnson, Nicholas, additional, LoRusso, Samantha, additional, Freimer, Miriam, additional, Shieh, Perry, additional, Subramony, S, additional, Leung, Doris, additional, van Engelen, Baziel, additional, Kools, Joost, additional, Dahlqvist-Leinhard, Olof, additional, Wildholm, Per, additional, Jiang, John, additional, Odueyungbo, Adefowope, additional, Tracewell, William, additional, Rojas, L. Alejandro, additional, Accorsi, Anthony, additional, Ronco, Lucienne, additional, Cadavid, Diego, additional, Moxham, Christopher, additional, Morabito, Christopher, additional, Gould, Robert, additional, and Mellion, Michelle, additional
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- 2022
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80. Reachable Workspace to Evaluate Efficacy of Losmapimod in Subjects with FSHD in Two Phase 2 Studies (P4-13.008)
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Mellion, Michelle, primary, Han, Jay, additional, Shoskes, Jennifer, additional, Tawil, Rabi, additional, Statland, Jeffrey, additional, Wang, Leo, additional, Genge, Angela, additional, Sacconi, Sabrina, additional, Lochmuller, Hanns, additional, Leiva, David Reyes, additional, Manera, Jordi Diaz, additional, Alonso-Perez, Jorge, additional, Gomez, Nuria Muelas, additional, Padilla, Juan Vilchez, additional, Pestronak, Alan, additional, Gibson, Summer, additional, Goyal, Namita, additional, Hamel, Johanna, additional, Hayward, Lawrence, additional, Johnson, Nicholas, additional, LoRusso, Samantha, additional, Freimer, Miriam, additional, Shieh, Perry, additional, Subramony, S, additional, Leung, Doris, additional, van Engelen, Baziel, additional, Kools, Joost, additional, Morabito, Christopher, additional, Jiang, John, additional, Tracewell, William, additional, Rojas, L. Alejandro, additional, and Accorsi, Anthony, additional
- Published
- 2022
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81. Whole Body MRI Quantitative muscle analysis to evaluate Efficacy of Losmapimod in a Phase 2 Placebo-Controlled Study in Subjects with FSHD (ReDUX4) (S23.009)
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Mellion, Michelle, primary, Tawil, Rabi, additional, Statland, Jeffrey, additional, Wang, Leo, additional, Genge, Angela, additional, Sacconi, Sabrina, additional, Lochmuller, Hanns, additional, Leiva, David Reyes, additional, Manera, Jordi Diaz, additional, Perez, Jorge Alonso, additional, Gomez, Nuria Muelas, additional, Padilla, Juan Vilchez, additional, Pestronk, Alan, additional, Gibson, Summer, additional, Goyal, Namita, additional, Hamel, Johanna, additional, Hayward, Lawrence, additional, Johnson, Nicholas, additional, LoRusso, Samantha, additional, Freimer, Miriam, additional, Shieh, Perry, additional, Subramony, S, additional, Leung, Doris, additional, van Engelen, Baziel, additional, Kools, Joost, additional, Leinhard, Olof Dahlqvist, additional, Widholm, Per, additional, Jiang, John, additional, Odueyungbo, Adefowope, additional, Tracewell, William, additional, Rojas, L. Alejandro, additional, Accorsi, Anthony, additional, Ronco, Lucienne, additional, Cadavid, Diego, additional, Moxham, Christopher, additional, Morabito, Christopher, additional, and Gould, Robert, additional
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- 2022
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82. Image: Global Shiftings in Media and Methods
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Gabriele Genge, Angela Stercken and Gabriele Genge, Angela Stercken
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- 2014
83. The importance of offering early genetic testing in everyone with amyotrophic lateral sclerosis
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Salmon, Kristiana, Kiernan, Matthew C., Kim, Seung H., Andersen, Peter M., Chio, Adriano, van den Berg, Leonard H., Van Damme, Philip, Al-Chalabi, Ammar, Lillo, Patricia, Andrews, Jinsy A., Genge, Angela, Salmon, Kristiana, Kiernan, Matthew C., Kim, Seung H., Andersen, Peter M., Chio, Adriano, van den Berg, Leonard H., Van Damme, Philip, Al-Chalabi, Ammar, Lillo, Patricia, Andrews, Jinsy A., and Genge, Angela
- Abstract
Several genetically-targeted therapies are being developed for ALS. Research is increasingly supportive of a greater incidence of clinically actionable variants in sporadic ALS than previously reported. Salmon et al. outline the need to improve access, and offer genetic testing to all people diagnosed with ALS.
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- 2022
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84. The importance of offering early genetic testing in everyone with amyotrophic lateral sclerosis
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Neurologen, Projectafdeling ALS, Brain, Regenerative Medicine and Stem Cells, Salmon, Kristiana, Kiernan, Matthew C, Kim, Seung H, Andersen, Peter M, Chio, Adriano, van den Berg, Leonard H, Van Damme, Philip, Al-Chalabi, Ammar, Lillo, Patricia, Andrews, Jinsy A, Genge, Angela, Neurologen, Projectafdeling ALS, Brain, Regenerative Medicine and Stem Cells, Salmon, Kristiana, Kiernan, Matthew C, Kim, Seung H, Andersen, Peter M, Chio, Adriano, van den Berg, Leonard H, Van Damme, Philip, Al-Chalabi, Ammar, Lillo, Patricia, Andrews, Jinsy A, and Genge, Angela
- Published
- 2022
85. A Phase 1 study of GDC-0134, a dual leucine zipper kinase inhibitor, in ALS
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Neurologen, Projectafdeling ALS, Brain, Regenerative Medicine and Stem Cells, Katz, Jonathan S, Rothstein, Jeffrey D, Cudkowicz, Merit E, Genge, Angela, Oskarsson, Björn, Hains, Avis B, Chen, Chen, Galanter, Joshua, Burgess, Braydon L, Cho, William, Kerchner, Geoffrey A, Yeh, Felix L, Ghosh, Arundhati Sengupta, Cheeti, Sravanthi, Brooks, Logan, Honigberg, Lee, Couch, Jessica A, Rothenberg, Michael E, Brunstein, Flavia, Sharma, Khema R, van den Berg, Leonard, Berry, James D, Glass, Jonathan D, Neurologen, Projectafdeling ALS, Brain, Regenerative Medicine and Stem Cells, Katz, Jonathan S, Rothstein, Jeffrey D, Cudkowicz, Merit E, Genge, Angela, Oskarsson, Björn, Hains, Avis B, Chen, Chen, Galanter, Joshua, Burgess, Braydon L, Cho, William, Kerchner, Geoffrey A, Yeh, Felix L, Ghosh, Arundhati Sengupta, Cheeti, Sravanthi, Brooks, Logan, Honigberg, Lee, Couch, Jessica A, Rothenberg, Michael E, Brunstein, Flavia, Sharma, Khema R, van den Berg, Leonard, Berry, James D, and Glass, Jonathan D
- Published
- 2022
86. International network for ALS research and care (INARC).
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Foucher, Juliette, Bunte, Tommy M., Bertone, Vanessa, Verschoor, Romy L., Couillard, Mathias, Straub, Corey, Genge, Angela, Ingre, Caroline, and van den Berg, Leonard H.
- Subjects
AMYOTROPHIC lateral sclerosis ,PROBLEM solving ,INTERNATIONAL cooperation ,RESEARCH teams ,PHYSICIANS - Abstract
The International Network for Amyotrophic Lateral Sclerosis (ALS) Research and Care (INARC) was founded in 2022. INARC's main goals are to offer a platform dedicated to staff members for ALS clinics and research teams who are not physicians. By nurturing experience and expertise exchanges to improve problem solving skills, the ultimate goal is to increase the standard ALS care and research. This brief report aims to describe the formation of INARC, the 2023 INARC meeting, as well as to report topics discussed, lessons learned and challenges raised by INARC members. [ABSTRACT FROM AUTHOR]
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- 2024
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87. Motor cortex functional connectivity is associated with underlying neurochemistry in ALS.
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Dey, Avyarthana, Luk, Collin C., Ishaque, Abdullah, Ta, Daniel, Srivastava, Ojas, Krebs, Dennell, Seres, Peter, Hanstock, Chris, Beaulieu, Christian, Korngut, Lawrence, Frayne, Richard, Zinman, Lorne, Graham, Simon, Genge, Angela, Briemberg, Hannah, Kalra, Sanjay, and Canadian ALS Neuroimaging Consortium (CALSNIC)
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MOTOR cortex ,FUNCTIONAL connectivity ,DIFFUSION magnetic resonance imaging ,NUCLEAR magnetic resonance spectroscopy ,AMYOTROPHIC lateral sclerosis - Abstract
Objective: To identify structural and neurochemical properties that underlie functional connectivity impairments of the primary motor cortex (PMC) and how these relate to clinical findings in amyotrophic lateral sclerosis (ALS).Methods: 52 patients with ALS and 52 healthy controls, matched for age and sex, were enrolled from 5 centres across Canada for the Canadian ALS Neuroimaging Consortium study. Resting-state functional MRI, diffusion tensor imaging and magnetic resonance spectroscopy data were acquired. Functional connectivity maps, diffusion metrics and neurometabolite ratios were obtained from the analyses of the acquired multimodal data. A clinical assessment of foot tapping (frequency) was performed to examine upper motor neuron function in all participants.Results: Compared with healthy controls, the primary motor cortex in ALS showed reduced functional connectivity with sensory (T=5.21), frontal (T=3.70), temporal (T=3.80), putaminal (T=4.03) and adjacent motor (T=4.60) regions. In the primary motor cortex, N-acetyl aspartate (NAA, a neuronal marker) ratios and diffusion metrics (mean, axial and radial diffusivity, fractional anisotropy (FA)) were altered. Within the ALS cohort, foot tapping frequency correlated with NAA (r=0.347) and white matter FA (r=0.537). NAA levels showed associations with disturbed functional connectivity of the motor cortex.Conclusion: In vivo neurochemistry may represent an effective imaging marker of impaired motor cortex functional connectivity in ALS. [ABSTRACT FROM AUTHOR]- Published
- 2023
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88. Oral edaravone demonstrated a favorable safety profile in patients with amyotrophic lateral sclerosis after 48 weeks of treatment.
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Genge, Angela, Pattee, Gary L., Sobue, Gen, Aoki, Masashi, Yoshino, Hiide, Couratier, Philippe, Lunetta, Christian, Petri, Susanne, Selness, Daniel, Bidani, Sachin, Hirai, Manabu, Sakata, Takeshi, Salah, Alejandro, Apple, Stephen, Wamil, Art, Kalin, Alexander, and Jackson, Carlayne E.
- Abstract
Introduction/Aims: An intravenous (IV) formulation of edaravone has been shown to slow the rate of physical functional decline in amyotrophic lateral sclerosis (ALS). An oral suspension formulation of edaravone was recently approved by the United States Food and Drug Administration for use in patients with ALS. This study assessed the safety and tolerability of oral edaravone. Methods: This global, open‐label, phase 3 study evaluated the long‐term safety and tolerability of oral edaravone in adults with ALS who had a baseline forced vital capacity ≥70% of predicted and disease duration ≤3 y. The primary safety analysis was assessed at weeks 24 and 48. Patients received a 105‐mg dose of oral edaravone in treatment cycles replicating the dosing of IV edaravone. Results: The study enrolled 185 patients (64.3% male; mean age, 59.9 y; mean disease duration, 1.56 y). The most common treatment‐emergent adverse events (TEAEs) at week 48 were fall (22.2%), muscular weakness (21.1%) and constipation (17.8%). Serious TEAEs were reported by 25.9% of patients; the most common were worsening ALS symptoms, dysphagia, dyspnea, and respiratory failure. Twelve TEAEs leading to death were reported. Forty‐six (24.9%) patients reported TEAEs that were considered related to study drug; the most common were fatigue, dizziness, headache, and constipation. Sixteen (8.6%) patients discontinued study drug due to TEAEs. No serious TEAEs were related to study drug. Discussion: This study indicated that oral edaravone was well tolerated during 48 wk of treatment, with no new safety concerns identified. [ABSTRACT FROM AUTHOR]
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- 2023
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89. CAPTURE ALS: the comprehensive analysis platform to understand, remedy and eliminate ALS.
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Picher-Martel, Vincent, Magnussen, Claire, Blais, Mathieu, Bubela, Tania, Das, Samir, Dionne, Annie, Evans, Alan C., Genge, Angela, Greiner, Russell, Iturria-Medina, Yasser, Johnston, Wendy, Jones, Kelvin, Kaneb, Hannah, Karamchandani, Jason, Moradipoor, Sara, Robertson, Janice, Rogaeva, Ekaterina, Taylor, David M., Vande Velde, Christine, and Yunusova, Yana
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VOICE disorders ,AMYOTROPHIC lateral sclerosis ,SCIENTIFIC community ,TREATMENT effectiveness ,THERAPEUTICS ,SPEECH - Abstract
The absence of disease modifying treatments for amyotrophic lateral sclerosis (ALS) is in large part a consequence of its complexity and heterogeneity. Deep clinical and biological phenotyping of people living with ALS would assist in the development of effective treatments and target specific biomarkers to monitor disease progression and inform on treatment efficacy. The objective of this paper is to present the Comprehensive Analysis Platform To Understand Remedy and Eliminate ALS (CAPTURE ALS), an open and translational platform for the scientific community currently in development. CAPTURE ALS is a Canadian-based platform designed to include participants' voices in its development and through execution. Standardized methods will be used to longitudinally characterize ALS patients and healthy controls through deep clinical phenotyping, neuroimaging, neurocognitive and speech assessments, genotyping and multisource biospecimen collection. This effort plugs into complementary Canadian and international initiatives to share common resources. Here, we describe in detail the infrastructure, operating procedures, and long-term vision of CAPTURE ALS to facilitate and accelerate translational ALS research in Canada and beyond. [ABSTRACT FROM AUTHOR]
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- 2023
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90. CAPTURE ALS: the comprehensive analysis platform to understand, remedy and eliminate ALS
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Picher-Martel, Vincent, primary, Magnussen, Claire, additional, Blais, Mathieu, additional, Bubela, Tania, additional, Das, Samir, additional, Dionne, Annie, additional, Evans, Alan C., additional, Genge, Angela, additional, Greiner, Russell, additional, Iturria-Medina, Yasser, additional, Johnston, Wendy, additional, Jones, Kelvin, additional, Kaneb, Hannah, additional, Karamchandani, Jason, additional, Moradipoor, Sara, additional, Robertson, Janice, additional, Rogaeva, Ekaterina, additional, Taylor, David M., additional, Vande Velde, Christine, additional, Yunusova, Yana, additional, Zinman, Lorne, additional, Kalra, Sanjay, additional, and Dupré, Nicolas, additional
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- 2022
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91. The importance of offering early genetic testing in everyone with amyotrophic lateral sclerosis
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Salmon, Kristiana, primary, Kiernan, Matthew C., additional, Kim, Seung H., additional, Andersen, Peter M., additional, Chio, Adriano, additional, van den Berg, Leonard H., additional, Van Damme, Philip, additional, Al-Chalabi, Ammar, additional, Lillo, Patricia, additional, Andrews, Jinsy A., additional, and Genge, Angela, additional
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- 2022
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92. A Phase 1 study of GDC ‐0134, a dual leucine zipper kinase inhibitor, in ALS
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Katz, Jonathan S., primary, Rothstein, Jeffrey D., additional, Cudkowicz, Merit E., additional, Genge, Angela, additional, Oskarsson, Björn, additional, Hains, Avis B., additional, Chen, Chen, additional, Galanter, Joshua, additional, Burgess, Braydon L., additional, Cho, William, additional, Kerchner, Geoffrey A., additional, Yeh, Felix L., additional, Ghosh, Arundhati Sengupta, additional, Cheeti, Sravanthi, additional, Brooks, Logan, additional, Honigberg, Lee, additional, Couch, Jessica A., additional, Rothenberg, Michael E., additional, Brunstein, Flavia, additional, Sharma, Khema R., additional, Berg, Leonard, additional, Berry, James D., additional, and Glass, Jonathan D., additional
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- 2022
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93. Distinct patterns of progressive gray and white matter degeneration in amyotrophic lateral sclerosis
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Ishaque, Abdullah, primary, Ta, Daniel, additional, Khan, Muhammad, additional, Zinman, Lorne, additional, Korngut, Lawrence, additional, Genge, Angela, additional, Dionne, Annie, additional, Briemberg, Hannah, additional, Luk, Collin, additional, Yang, Yee‐Hong, additional, Beaulieu, Christian, additional, Emery, Derek, additional, Eurich, Dean T., additional, Frayne, Richard, additional, Graham, Simon, additional, Wilman, Alan, additional, Dupré, Nicolas, additional, and Kalra, Sanjay, additional
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- 2021
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94. Complement Inhibition in Myasthenia Gravis and Neuromyelitis Optica Spectrum Disorder
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Dodig, Dubravka, primary, Genge, Angela, additional, Selchen, Daniel, additional, and Freedman, Mark S., additional
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- 2021
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95. MN-166 (ibudilast) in amyotrophic lateral sclerosis in a Phase IIb/III study: COMBAT-ALS study design
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Oskarsson, Björn, primary, Maragakis, Nicholas, additional, Bedlack, Richard S, additional, Goyal, Namita, additional, Meyer, Jenny A, additional, Genge, Angela, additional, Bodkin, Cynthia, additional, Maiser, Samuel, additional, Staff, Nathan, additional, Zinman, Lorne, additional, Olney, Nicholas, additional, Turnbull, John, additional, Brooks, Benjamin Rix, additional, Klonowski, Emelia, additional, Makhay, Malath, additional, Yasui, Seiichi, additional, and Matsuda, Kazuko, additional
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- 2021
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96. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial
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Diaz-Manera, Jordi, primary, Kishnani, Priya S, additional, Kushlaf, Hani, additional, Ladha, Shafeeq, additional, Mozaffar, Tahseen, additional, Straub, Volker, additional, Toscano, Antonio, additional, van der Ploeg, Ans T, additional, Berger, Kenneth I, additional, Clemens, Paula R, additional, Chien, Yin-Hsiu, additional, Day, John W, additional, Illarioshkin, Sergey, additional, Roberts, Mark, additional, Attarian, Shahram, additional, Borges, Joao Lindolfo, additional, Bouhour, Francoise, additional, Choi, Young Chul, additional, Erdem-Ozdamar, Sevim, additional, Goker-Alpan, Ozlem, additional, Kostera-Pruszczyk, Anna, additional, Haack, Kristina An, additional, Hug, Christopher, additional, Huynh-Ba, Olivier, additional, Johnson, Judith, additional, Thibault, Nathan, additional, Zhou, Tianyue, additional, Dimachkie, Mazen M, additional, Schoser, Benedikt, additional, Behin, Anthony, additional, Boentert, Matthias, additional, Carvalho, Gerson, additional, Chahin, Nizar, additional, Charrow, Joel, additional, Deegan, Patrick, additional, Durmus Tekce, Hacer, additional, Duval, Fanny, additional, Genge, Angela, additional, Gutmann, Ludwig, additional, Henderson, Robert D, additional, Hennermann, Julia B, additional, Hiwot, Tarekegn, additional, Hughes, Derralynn, additional, Karaa, Amel, additional, Karam, Chafic, additional, Kautzky-Willer, Alexandra, additional, Komaki, Hirofumi, additional, Laforet, Pascal, additional, Longo, Nicola, additional, Malinova, Vera, additional, Maré, Ricardo, additional, Maxit, Clarisa, additional, Mengel, Eugen, additional, Moggio, Maurizio Gualtiero, additional, Molnár, Mária Judit, additional, Mongini, Tiziana Enrica, additional, Nadaj-Pakleza, Aleksandra, additional, Nascimento Osorio, Andres, additional, Noury, Jean-Baptiste, additional, Oliveira, Acary Souza Bulle, additional, Parman, Yesim, additional, Pena, Loren, additional, Remiche, Gauthier, additional, Sciacco, Monica, additional, Shieh, Perry B, additional, Smith, Cheryl, additional, Stulnig, Thomas, additional, Taithe, Frederic, additional, Tard, Céline, additional, Tarnopolsky, Mark, additional, Vorgerd, Matthias, additional, Whitley, Chester, additional, Young, Peter, additional, Alonso-Pérez, Jorge, additional, Altemus, Patricia, additional, Aubé-Nathier, Anne-Catherine, additional, Avelar, Jennifer B, additional, Bailey, Carrie, additional, Bekircan-Kurt, Can Ebru, additional, Billy, Jenny, additional, Boschi, Silvia, additional, Brown, Kathryn E, additional, Carrera Garcia, Laura, additional, Chase, Lauren, additional, Cirne, Hamilton, additional, Danjoux, Loïc, additional, Davion, Jean-Baptiste, additional, DeArmey, Stephanie, additional, Fedotova, Ekaterina, additional, Gandolfo, Eve, additional, Grosz, Zoltan, additional, Guellec, Dewi, additional, Guettsches, Anne-Katrin, additional, Guglieri, Michela, additional, Hatcher, Erin, additional, Helms, Sina, additional, Hufgard-Leitner, Miriam, additional, Klyushnikov, Sergey A., additional, Langton, Jacqui, additional, Linková, Lenka, additional, Mavroudakis, Nicolas, additional, Mazurová, Stella, additional, Mori, Madoka, additional, Müller-Miny, Louisa, additional, Musumeci, Olimpia, additional, Nance, Christopher S, additional, Natera-de Benito, Daniel, additional, Neel, Robert, additional, Niizawa, Gabriela A, additional, Noll, Lauren, additional, Ortega, Erik, additional, Pasnoor, Mamatha, additional, Pautot, Vivien, additional, Potulska-Chromik, Anna, additional, Pugliese, Alessia, additional, Questienne, Claire, additional, Ramos Lopes, Margarida, additional, Reyes-Leiva, David, additional, Riedl, Michaela, additional, Rugiero, Marcelo Francisco, additional, Salort-Campana, Emmanuelle, additional, Sgobbi Souza, Paulo Victor, additional, Sole, Guilhem, additional, Solera, Luca, additional, Souto Lopes, Suzara, additional, Specht, Sabine, additional, Statland, Jeffrey, additional, Swenson, Andrea, additional, Tan, Chong Yew, additional, Tizon, Sónia, additional, van der Beek, N A M E, additional, van Kooten, Harmke A., additional, Wencel, Marie, additional, Wenninger, Stephan, additional, and Zagnoli, Fabien, additional
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- 2021
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97. Defining cognitive impairment in amyotrophic lateral sclerosis: an evaluation of empirical approaches.
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McMillan, Corey T., Wuu, Joanne, Rascovsky, Katya, Cosentino, Stephanie, Grossman, Murray, Elman, Lauren, Quinn, Colin, Rosario, Luis, Stark, Jessica H., Granit, Volkan, Briemberg, Hannah, Chenji, Sneha, Dionne, Annie, Genge, Angela, Johnston, Wendy, Korngut, Lawrence, Shoesmith, Christen, Zinman, Lorne, Kalra, Sanjay, and Benatar, Michael
- Subjects
AMYOTROPHIC lateral sclerosis ,COGNITION disorders ,NEURODEGENERATION ,REFERENCE values ,QUANTILE regression - Abstract
Objective: Amyotrophic lateral sclerosis (ALS) is a multi-system disorder characterized primarily by motor neuron degeneration, but may be accompanied by cognitive dysfunction. Statistically appropriate criteria for establishing cognitive impairment (CI) in ALS are lacking. We evaluate quantile regression (QR), that accounts for age and education, relative to a traditional two standard deviation (SD) cutoff for defining CI. Methods: QR of cross-sectional data from a multi-center North American Control (NAC) cohort of 269 healthy adults was used to model the 5
th percentile of cognitive scores on the Edinburgh Cognitive and Behavioral ALS Screen (ECAS). The QR approach was compared to traditional two SD cutoff approach using the same NAC cohort (2SD-NAC) and to existing UK-based normative data derived using the 2SD approach (2SD-UK) to assess the impact of cohort selection and statistical model in identifying CI in 182 ALS patients. Results: QR-NAC models revealed that age and education impact cognitive performance on the ECAS. Based on QR-NAC normative cutoffs, the frequency of CI in the 182 PENN ALS patients was 15.9% for ALS specific, 12.6% for ALS nonspecific, and 15.4% for ECAS total. This frequency of CI is substantially more conservative in comparison to the 2SD-UK (20.3%–34.6%) and modestly more conservative to the 2SD-NAC (14.3%–16.5%) approaches for estimating CI. Conclusions: The choice of normative cohort has a substantial impact and choice of statistical method a modest impact on defining CI in ALS. This report establishes normative ECAS thresholds to identify whether ALS patients in the North American population have CI. [ABSTRACT FROM AUTHOR]- Published
- 2022
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98. Safety and efficacy of oral levosimendan in people with amyotrophic lateral sclerosis (the REFALS study): a randomised, double-blind, placebo-controlled phase 3 trial
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Cudkowicz, Merit, primary, Genge, Angela, additional, Maragakis, Nicholas, additional, Petri, Susanne, additional, van den Berg, Leonard, additional, Aho, Valtteri V, additional, Sarapohja, Toni, additional, Kuoppamäki, Mikko, additional, Garratt, Chris, additional, Al-Chalabi, Ammar, additional, Kiernan, Matthew, additional, Mathers, Susan, additional, Henderson, Robert, additional, Needham, Merrilee, additional, Schultz, David, additional, Löscher, Wolfgang, additional, Mitrovic, Nenad, additional, Rath, Jakob, additional, Damme, Philip Van, additional, De Bleecker, Jan L., additional, Delstanche, Stéphanie, additional, Johnston, Wendy, additional, Zinman, Lorne, additional, O'Connell, Colleen, additional, Matte, Genevieve, additional, Dionne, Annie, additional, Korngut, Lawrence, additional, Turnbull, John, additional, Laaksovirta, Hannu, additional, Jokela, Manu, additional, Tapiola, Tero, additional, Soriani, Marie-Hélène, additional, Couratier, Philippe, additional, Camu, William, additional, Corcia, Philippe, additional, Ludolph, Albert, additional, Großkreutz, Julian, additional, Meyer, Thomas, additional, Boentert, Matthias, additional, Schrank, Berthold, additional, Prudlo, Johannes, additional, Untucht, Robert, additional, Hardiman, Orla, additional, Siciliano, Gabriele, additional, Chio', Adriano, additional, Mazzini, Letizia, additional, Inghilleri, Maurizio, additional, Caponnetto, Claudia, additional, Mora, Gabriele, additional, Mora Pardina, Jesús S, additional, Farrero Munoz, Eva, additional, Vázquez Costa, Juan F, additional, Aguera Morales, Eduardo, additional, Varona, Luis, additional, Andersen, Peter, additional, Ingre, Caroline, additional, Johansson, Rune, additional, Radunovic, Aleksandar, additional, Young, Carolyn, additional, Babu, Suma, additional, Shaibani, Aziz, additional, Staff, Nathan, additional, Vu, Tuan, additional, Rivner, Michael, additional, Scelsa, Stephen, additional, Sivakumar, Kumaraswamy, additional, Waheed, Waqar, additional, Heitzman, Daragh, additional, Rana, Sandeep, additional, Pattee, Gary, additional, Ajroud-Driss, Senda, additional, Bayat, Elham, additional, Kasarskis, Edward, additional, Lange, Dale J, additional, Elliott, Michael, additional, Harris, Brent, additional, Felice, Kevin, additional, Pulley, Michael T, additional, Kwan, Justin, additional, Brown, Martin, additional, Ravits, John, additional, Burford, Matthew, additional, Karam, Chafic, additional, Miller, Timothy, additional, Andrews, Jinsy, additional, Levine, Todd, additional, Locatelli, Eduardo, additional, Wymer, James, additional, Bedlack, Richard, additional, Fee, Dominic, additional, Goyal, Namita, additional, Oskarsson, Bjorn, additional, McCluskey, Leo, additional, Caress, James, additional, Weiss, Michael, additional, Quick, Adam, additional, Bromberg, Mark, additional, Lacomis, David, additional, Goutman, Stephen, additional, Rezania, Kourosh, additional, Guliani, Gaurav, additional, Goslin, Kimberly, additional, and Katz, Jonathan S, additional
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- 2021
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99. Genetic testing for amyotrophic lateral sclerosis in Canada – an assessment of current practices
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Salmon, Kristiana, primary, Anoja, Nancy, additional, Breiner, Ari, additional, Chum, Marvin, additional, Dionne, Annie, additional, Dupré, Nicolas, additional, Fiander, Amanda, additional, Fok, Daniel, additional, Ghavanini, Amer, additional, Gosselin, Sylvie, additional, Izenberg, Aaron, additional, Johnston, Wendy, additional, Kalra, Sanjay, additional, Matte, Geneviève, additional, Melanson, Michel, additional, O’Connell, Colleen, additional, Ritsma, Benjamin, additional, Schellenberg, Kerri, additional, Shoesmith, Christen, additional, Tremblay, Sandra, additional, Williams, Heather, additional, and Genge, Angela, additional
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- 2021
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100. A Road Map for Remote Digital Health Technology for Motor Neuron Disease
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van Eijk, Ruben P A, primary, Beelen, Anita, additional, Kruitwagen, Esther T, additional, Murray, Deirdre, additional, Radakovic, Ratko, additional, Hobson, Esther, additional, Knox, Liam, additional, Helleman, Jochem, additional, Burke, Tom, additional, Rubio Pérez, Miguel Ángel, additional, Reviers, Evy, additional, Genge, Angela, additional, Steyn, Frederik J, additional, Ngo, Shyuan, additional, Eaglesham, John, additional, Roes, Kit C B, additional, van den Berg, Leonard H, additional, Hardiman, Orla, additional, and McDermott, Christopher J, additional
- Published
- 2021
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