Progressive Neurochemical Abnormalities in Cognitive and Motor Subgroups of Amyotrophic Lateral Sclerosis: A Prospective Multicenter Study von Ta, Daniel, Ishaque, Abdullah, Srivastava, Ojas, Hanstock, Chris, Seres, Peter, Eurich, Dean T., Luk, Collin, Briemberg, Hannah, Frayne, Richard, Genge, Angela L., Graham, Simon J., Korngut, Lawrence, Zinman, Lorne, Kalra, Sanjay

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Investigating Late-Onset Pompe Prevalence in Neuromuscular Medicine Academic Practices: The IPaNeMA Study von Wencel, Marie, Shaibani, Aziz, Goyal, Namita A., Dimachkie, Mazen M., Trivedi, Jaya, Johnson, Nicholas E., Gutmann, Laurie, Wicklund, Matthew P., Bandyopadhay, Sankar, Genge, Angela L., Freimer, Miriam L., Goyal, Neelam, Pestronk, Alan, Florence, Julaine, Karam, Chafic, Ralph, Jeffrey W., Rasheed, Zinah, Hays, Melissa, Hopkins, Steve, Mozaffar, Tahseen

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Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS von Miller, Timothy, Cudkowicz, Merit, Shaw, Pamela J, Andersen, Peter M, Atassi, Nazem, Bucelli, Robert C, Genge, Angela, Glass, Jonathan, Ladha, Shafeeq, Ludolph, Albert L, Maragakis, Nicholas J, McDermott, Christopher J, Pestronk, Alan, Ravits, John, Salachas, François, Trudell, Randall, Van Damme, Philip, Zinman, Lorne, Bennett, C. Frank, Lane, Roger, Sandrock, Alfred, Runz, Heiko, Graham, Danielle, Houshyar, Hani, McCampbell, Alexander, Nestorov, Ivan, Chang, Ih, McNeill, Manjit, Fanning, Laura, Fradette, Stephanie, Ferguson, Toby A

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Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Rando... von Howard, James F, Nowak, Richard J, Wolfe, Gil I, Freimer, Miriam L, Vu, Tuan H, Hinton, John L, Benatar, Michael, Duda, Petra W, MacDougall, James E, Farzaneh-Far, Ramin, Kaminski, Henry J, Barohn, Richard, Dimachkie, Mazen, Pasnoor, Mamatha, Farmakidis, Constantine, Liu, Tina, Colgan, Samantha, Benatar, Michael G, Bertorini, Tulio, Pillai, Rekha, Henegar, Robert, Bromberg, Mark, Gibson, Summer, Janecki, Teresa, Freimer, Miriam, Elsheikh, Bakri, Matisak, Paige, Genge, Angela, Guidon, Amanda, David, William, Habib, Ali A, Mathew, Veena, Mozaffar, Tahseen, Hewitt, William, Barnett, Deborah, Sullivan, Patricia, Ho, Doreen, Traub, Rebecca E, Chopra, Manisha, Aly, Radwa, Bayat, Elham, Abu-Rub, Mohammad, Khan, Shaida, Lange, Dale, Holzberg, Shara, Khatri, Bhupendra, Lindman, Emily, Olapo, Tayo, Sershon, Lisa M, Lisak, Robert P, Bernitsas, Evanthia, Jia, Kelly, Malik, Rabia, Lewis-Collins, Tiffany D, Nicolle, Michael, Sharma, Aditi, Roy, Bhaskar, Nye, Joan, Pulley, Michael, Berger, Alan, Shabbir, Yasmeen, Sachdev, Amit, Patterson, Kimberly, Siddiqi, Zaeem, Sivak, Mark, Bratton, Joan, Small, George, Kohli, Anem, Fetter, Mary, Vu, Tuan, Lam, Lucy, Harvey, Brittany, Silvestri, Nicholas, Patrick, Kara, Zakalik, Karen, MacDougall, James, Pontius, Angela, Hoarty, Michelle

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International network for ALS research and care (INARC) von Foucher, J, Bunte, TM, Bertone, V, Verschoor, RL, Couillard, M, Straub, C, Genge, A, Ingre, C, van den Berg, LH

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Phase 3, open-label, multicenter safety study of oral edaravone in patients with amyotrophic lateral sclerosis (MT-1186-A01): 48-week results von Genge, Angela, Pattee, Gary L., Sobue, Gen, Couratier, Philippe, Selness, Daniel, Bidani, Sachin, Hirai, Manabu, Salah, Alejandro, Apple, Stephen

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Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel von Tarnopolsky, Mark, Katzberg, Hans, Petrof, Basil J., Sirrs, Sandra, Sarnat, Harvey B., Myers, Kimberley, Dupré, Nicolas, Dodig, Dubravka, Genge, Angela, Venance, Shannon L., Korngut, Lawrence, Raiman, Julian, Khan, Aneal

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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) von Genge, Angela, Pattee, Gary L., Sobue, Gen, Couratier, Philippe, Selness, Daniel, Hirai, Manabu, Sakata, Takeshi, Salah, Alejandro, Apple, Stephen

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Validating Automatic Diadochokinesis Analysis Methods across Dysarthria Severity and Syllable Task in Amyotrophic Lateral Sclerosis von Tanchip, Chelsea, Guarin, Diego L, McKinlay, Scotia, Barnett, Carolina, Kalra, Sanjay, Genge, Angela, Korngut, Lawrence, Green, Jordan R, Berry, James, Zinman, Lorne, Yadollahi, Azadeh, Abrahao, Agessandro, Yunusova, Yana

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Oral edaravone demonstrated a favorable safety profile in patients with amyotrophic lateral sclerosis after 48 weeks of treatment von 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, Jackson, Carlayne E.

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Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blind... von van den Berg, Leonard H, Rothstein, Jeffrey D, Shaw, Pamela J, Babu, Suma, Benatar, Michael, Bucelli, Robert C, Genge, Angela, Glass, Jonathan D, Hardiman, Orla, Libri, Vincenzo, Mobach, Theodore, Oskarsson, Björn, Pattee, Gary L, Ravits, John, Shaw, Christopher E, Weber, Markus, Zinman, Lorne, Jafar-nejad, Paymaan, Rigo, Frank, Lin, Luan, Ferguson, Toby A, Gotter, Anthony L, Graham, Danielle, Monine, Michael, Inra, Jennifer, Sinks, Susie, Eraly, Satish, Garafalo, Steve, Fradette, Stephanie

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A Phase 1 study of GDC‐0134, a dual leucine zipper kinase inhibitor, in ALS von 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., Berg, Leonard, Berry, James D., Glass, Jonathan D.

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COURAGE-ALS: a randomized, double-blind phase 3 study designed to improve participant experience and increase the probability of success von Shefner, JM, Al-Chalabi, A, Andrews, JA, Chio, A, De Carvalho, M, Cockroft, BM, Corcia, P, Couratier, P, Cudkowicz, ME, Genge, A, Hardiman, O, Heiman-Patterson, T, Henderson, RD, Ingre, C, Jackson, CE, Johnston, W, Lechtzin, N, Ludolph, A, Maragakis, NJ, Miller, TM, Pardina, JMS, Petri, S, Simmons, Z, Van Den Berg, L, Zinman, L, Kupfer, S, Malik, FI, Meng, L, Simkins, TJ, Wei, J, Wolff, AA, Rudnicki, SA

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A randomized controlled trial of methotrexate for patients with generalized myasthenia gravis von Pasnoor, Mamatha, He, Jianghua, Herbelin, Laura, Burns, Ted M, Nations, Sharon, Bril, Vera, Wang, Annabel K, Elsheikh, Bakri H, Kissel, John T, Saperstein, David, Shaibani, J Aziz, Jackson, Carlayne, Swenson, Andrea, Howard, James F, Goyal, Namita, David, William, Wicklund, Matthew, Pulley, Michael, Becker, Mara, Mozaffar, Tahseen, Benatar, Michael, Pazcuzzi, Robert, Simpson, Ericka, Rosenfeld, Jeffrey, Dimachkie, Mazen M, Statland, Jeffrey M, Barohn, Richard J

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Oral edaravone demonstrated a favorable safety profile in patients with amyotrophic lateral sclerosis after 48weeks of treatment von 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, Jackson, Carlayne E

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Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study von Rudnicki, Stacy A., Andrews, Jinsy A., Duong, Tina, Cockroft, Bettina M., Malik, Fady I., Meng, Lisa, Wei, Jenny, Wolff, Andrew A., Genge, Angela, Johnson, Nicholas E., Tesi-Rocha, Carolina, Connolly, Anne M., Darras, Basil T., Felice, Kevin, Finkel, Richard S., Shieh, Perry B., Mah, Jean K., Statland, Jeffrey, Campbell, Craig, Habib, Ali A., Kuntz, Nancy L., Oskoui, Maryam, Day, John W.

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Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial von Tawil, Rabi, Wagner, Kathryn R, Hamel, Johanna I, Leung, Doris G, Statland, Jeffrey M, Wang, Leo H, Genge, Angela, Sacconi, Sabrina, Lochmüller, Hanns, Reyes-Leiva, David, Diaz-Manera, Jordi, Alonso-Perez, Jorge, Muelas, Nuria, Vilchez, Juan J, Pestronk, Alan, Gibson, Summer, Goyal, Namita A, Hayward, Lawrence J, Johnson, Nicholas, LoRusso, Samantha, Freimer, Miriam, Shieh, Perry B, Subramony, S H, van Engelen, Baziel, Kools, Joost, Leinhard, Olof Dahlqvist, Widholm, Per, Morabito, Christopher, Moxham, Christopher M, Cadavid, Diego, Mellion, Michelle L, Odueyungbo, Adefowope, Tracewell, William G, Accorsi, Anthony, Ronco, Lucienne, Gould, Robert J, Shoskes, Jennifer, Rojas, Luis Alejandro, Jiang, John G

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Oral fingolimod for chronic inflammatory demyelinating polyradiculoneuropathy (FORCIDP Trial): a double-blind, multicentre, randomised controlled trial von Hughes, Richard, Dalakas, Marinos C, Merkies, Ingemar, Latov, Norman, Léger, Jean-Marc, Nobile-Orazio, Eduardo, Sobue, Gen, Genge, Angela, Cornblath, David, Merschhemke, Martin, Ervin, Carolyn Marie, Agoropoulou, Catherine, Hartung, Hans-Peter, Day, Timothy, Spies, Judith, Roberts, Leslie, Van Damme, Philip, Van den Bergh, Peter YK, Maertens de Noordhout, Alain, Dionne, Annie, Larue, Sandrine, Massie, Rami, Melanson, Michel, Camu, William, De Seze, Jérôme, Le Masson, Gwendal, Pouget, Jean, Schmidt, Jens, Kimiskidis, Vasilios K, Chapman, Joab, Drory, Vivian E, Fazio, Raffaella, Gallia, Francesca, Kusunoki, Susumu, Mori, Masahiro, Iijima, Masahiro, Okamoto, Tomoko, Baba, Masayuki, Faber, Catharina G, van Schaik, Ivo N, Fryze, Waldemar, Motta, Ewa, Selmaj, Krzysztof, Casasnovas, Carlos, Sola, Antonio Guerrero, Illa, Isabel, Holt, James, Miller, James AL, Lunn, Michael P, Brannagan, Thomas H, Brown, Martin, Kelemen, John, Iyadurai, Stanley, Rezania, Kourosh, Sharma, Khema R, Tandan, Rup, Gudesblatt, Mark, Lawson, Victoria, Amato, Anthony A

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Reachable Workspace to Evaluate Efficacy of Losmapimod in Subjects with FSHD in Two Phase 2 Studies (P4-13.008) von Mellion, Michelle, Han, Jay, Shoskes, Jennifer, Tawil, Rabi, Statland, Jeffrey, Wang, Leo, Genge, Angela, Sacconi, Sabrina, Lochmuller, Hanns, Leiva, David Reyes, Manera, Jordi Diaz, Alonso-Perez, Jorge, Gomez, Nuria Muelas, Padilla, Juan Vilchez, Pestronak, Alan, Gibson, Summer, Goyal, Namita, Hamel, Johanna, Hayward, Lawrence, Johnson, Nicholas, LoRusso, Samantha, Freimer, Miriam, Shieh, Perry, Subramony, S, Leung, Doris, van Engelen, Baziel, Kools, Joost, Morabito, Christopher, Jiang, John, Tracewell, William, Rojas, L. Alejandro, Accorsi, Anthony

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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) von Tawil, Rabi, Statland, Jeffrey, Wang, Leo, Genge, Angela, Sacconi, Sabrina, Lochmuller, Hanns, Leiva, David Reyes, Manera, Jordi Diaz, Alonso-Perez, Jorge, Gomez, Nuria Muelas, Padilla, Juan Vilchez, Pestronk, Alan, Gibson, Summer, Goyal, Namita, Hamel, Johanna, Hayward, Lawrence, Johnson, Nicholas, LoRusso, Samantha, Freimer, Miriam, Shieh, Perry, Subramony, S, Leung, Doris, van Engelen, Baziel, Kools, Joost, Dahlqvist-Leinhard, Olof, Wildholm, Per, Jiang, John, Odueyungbo, Adefowope, Tracewell, William, Rojas, L. Alejandro, Accorsi, Anthony, Ronco, Lucienne, Cadavid, Diego, Moxham, Christopher, Morabito, Christopher, Gould, Robert, Mellion, Michelle

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