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2. SF2Former: Amyotrophic Lateral Sclerosis Identification From Multi-center MRI Data Using Spatial and Frequency Fusion Transformer

Author

Kushol, Rafsanjany, Luk, Collin C., Dey, Avyarthana, Benatar, Michael, Briemberg, Hannah, Dionne, Annie, Dupré, Nicolas, Frayne, Richard, Genge, Angela, Gibson, Summer, Graham, Simon J., Korngut, Lawrence, Seres, Peter, Welsh, Robert C., Wilman, Alan, Zinman, Lorne, Kalra, Sanjay, and Yang, Yee-Hong

Subjects
Electrical Engineering and Systems Science - Image and Video Processing, Computer Science - Computer Vision and Pattern Recognition, Computer Science - Machine Learning
Abstract

Amyotrophic Lateral Sclerosis (ALS) is a complex neurodegenerative disorder involving motor neuron degeneration. Significant research has begun to establish brain magnetic resonance imaging (MRI) as a potential biomarker to diagnose and monitor the state of the disease. Deep learning has turned into a prominent class of machine learning programs in computer vision and has been successfully employed to solve diverse medical image analysis tasks. However, deep learning-based methods applied to neuroimaging have not achieved superior performance in ALS patients classification from healthy controls due to having insignificant structural changes correlated with pathological features. Therefore, the critical challenge in deep models is to determine useful discriminative features with limited training data. By exploiting the long-range relationship of image features, this study introduces a framework named SF2Former that leverages vision transformer architecture's power to distinguish the ALS subjects from the control group. To further improve the network's performance, spatial and frequency domain information are combined because MRI scans are captured in the frequency domain before being converted to the spatial domain. The proposed framework is trained with a set of consecutive coronal 2D slices, which uses the pre-trained weights on ImageNet by leveraging transfer learning. Finally, a majority voting scheme has been employed to those coronal slices of a particular subject to produce the final classification decision. Our proposed architecture has been thoroughly assessed with multi-modal neuroimaging data using two well-organized versions of the Canadian ALS Neuroimaging Consortium (CALSNIC) multi-center datasets. The experimental results demonstrate the superiority of our proposed strategy in terms of classification accuracy compared with several popular deep learning-based techniques., Comment: 17 pages, 8 figures

Published
2023
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3. Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study

Author

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, and Fradette, Stephanie

Published
2024
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4. Randomized phase 2 study of ACE-083, a muscle-promoting agent, in facioscapulohumeral muscular dystrophy.

Author

Statland, Jeffrey, Campbell, Craig, Desai, Urvi, Karam, Chafic, Díaz-Manera, Jordi, Guptill, Jeffrey, Korngut, Lawrence, Genge, Angela, Tawil, Rabi, Elman, Lauren, Joyce, Nanette, Wagner, Kathryn, Manousakis, Georgios, Amato, Anthony, Butterfield, Russell, Shieh, Perry, Wicklund, Matthew, Gamez, Josep, Bodkin, Cynthia, Pestronk, Alan, Weihl, Conrad, Vilchez-Padilla, Juan, Johnson, Nicholas, Mathews, Katherine, Miller, Barry, Leneus, Ashley, Fowler, Marcie, van de Rijn, Marc, and Attie, Kenneth

Subjects
FSHD, controlled trial, facioscapulohumeral muscular dystrophy, randomized, Adolescent, Adult, Cytomegalovirus Infections, Humans, Magnetic Resonance Imaging, Muscle Contraction, Muscle, Skeletal, Muscular Dystrophy, Facioscapulohumeral
Abstract

INTRODUCTION/AIMS: Facioscapulohumeral muscular dystrophy (FSHD) is a slowly progressive muscular dystrophy without approved therapies. In this study we evaluated whether locally acting ACE-083 could safely increase muscle volume and improve functional outcomes in adults with FSHD. METHODS: Participants were at least 18 years old and had FSHD1/FSHD2. Part 1 was open label, ascending dose, assessing safety and tolerability (primary objective). Part 2 was randomized, double-blind for 6 months, evaluating ACE-083240 mg/muscle vs placebo injected bilaterally every 3 weeks in the biceps brachii (BB) or tibialis anterior (TA) muscles, followed by 6 months of open label. Magnetic resonance imaging measures included total muscle volume (TMV; primary objective), fat fraction (FF), and contractile muscle volume (CMV). Functional measures included 6-minute walk test, 10-meter walk/run, and 4-stair climb (TA group), and performance of upper limb midlevel/elbow score (BB group). Strength, patient-reported outcomes (PROs), and safety were also evaluated. RESULTS: Parts 1 and 2 enrolled 37 and 58 participants, respectively. Among 55 participants evaluable in Part 2, the least-squares mean (90% confidence interval, analysis of covariance) treatment difference for TMV was 16.4% (9.8%-23.0%) in the BB group (P

Published
2022

5. The neurophysiological brain-fingerprint of Parkinson’s disease

Author

Breitner, John, Poirier, Judes, Baillet, Sylvain, Bellec, Pierre, Bohbot, Véronique, Chakravarty, Mallar, Collins, Louis, Etienne, Pierre, Evans, Alan, Gauthier, Serge, Hoge, Rick, Ituria-Medina, Yasser, Multhaup, Gerhard, Münter, Lisa-Marie, Rajah, Natasha, Rosa-Neto, Pedro, Soucy, Jean-Paul, Vachon-Presseau, Etienne, Villeneuve, Sylvia, Amouyel, Philippe, Appleby, Melissa, Ashton, Nicholas, Auld, Daniel, Ayranci, Gülebru, Bedetti, Christophe, Beland, Marie-Lise, Blennow, Kaj, Westman, Ann Brinkmalm, Cuello, Claudio, Dadar, Mahsa, Daoust, Leslie-Ann, Das, Samir, Dauar-Tedeschi, Marina, De Beaumont, Louis, Dea, Doris, Descoteaux, Maxime, Dufour, Marianne, Farzin, Sarah, Ferdinand, Fabiola, Fonov, Vladimir, Gonneaud, Julie, Kat, Justin, Kazazian, Christina, Labonté, Anne, Lafaille-Magnan, Marie-Elyse, Lalancette, Marc, Lambert, Jean-Charles, Leoutsakos, Jeannie-Marie, Mahar, Laura, Mathieu, Axel, McSweeney, Melissa, Meyer, Pierre-François, Miron, Justin, Near, Jamie, NewboldFox, Holly, Nilsson, Nathalie, Orban, Pierre, Picard, Cynthia, Binette, Alexa Pichet, Poline, Jean-Baptiste, Rabipour, Sheida, Salaciak, Alyssa, Settimi, Matthew, Subramaniapillai, Sivaniya, Tam, Angela, Tardif, Christine, Théroux, Louise, Tremblay-Mercier, Jennifer, Tullo, Stephanie, Ulku, Irem, Vallée, Isabelle, Zetterberg, Henrik, Nair, Vasavan, Pruessner, Jens, Aisen, Paul, Anthal, Elena, Barkun, Alan, Beaudry, Thomas, Benbouhoud, Fatiha, Brandt, Jason, Carmo, Leopoldina, Carrier, Charles Edouard, Cheewakriengkrai, Laksanun, Courcot, Blandine, Couture, Doris, Craft, Suzanne, Dansereau, Christian, Debacker, Clément, Desautels, René, Dubuc, Sylvie, Duclair, Guerda, Eisenberg, Mark, El-Khoury, Rana, Faubert, Anne-Marie, Fontaine, David, Frappier, Josée, Frenette, Joanne, Gagné, Guylaine, Gervais, Valérie, Giles, Renuka, Gordon, Renee, Jack, Clifford, Jutras, Benoit, Khachaturian, Zaven, Knopman, David, Kostopoulos, Penelope, Lapalme, Félix, Lee, Tanya, Lepage, Claude, Leppert, Illana, Madjar, Cécile, Maillet, David, Maltais, Jean-Robert, Mathotaarachchi, Sulantha, Mayrand, Ginette, Michaud, Diane, Montine, Thomas, Morris, John, Pagé, Véronique, Pascoal, Tharick, Peillieux, Sandra, Petkova, Mirela, Pogossova, Galina, Rioux, Pierre, Sager, Mark, Saint-Fort, Eunice Farah, Savard, Mélissa, Sperling, Reisa, Tabrizi, Shirin, Tariot, Pierre, Teigner, Eduard, Thomas, Ronald, Toussaint, Paule-Joanne, Tuwaig, Miranda, Venugopalan, Vinod, Verfaillie, Sander, Vogel, Jacob, Wan, Karen, Wang, Seqian, Yu, Elsa, Beaulieu-Boire, Isabelle, Blanchet, Pierre, Bogard, Sarah, Bouchard, Manon, Chouinard, Sylvain, Cicchetti, Francesca, Cloutier, Martin, Dagher, Alain, Degroot, Clotilde, Desautels, Alex, Dion, Marie Hélène, Drouin-Ouellet, Janelle, Dufresne, Anne-Marie, Dupré, Nicolas, Duquette, Antoine, Durcan, Thomas, Fellows, Lesley K., Fon, Edward, Gagnon, Jean-François, Gan-Or, Ziv, Genge, Angela, Jodoin, Nicolas, Karamchandani, Jason, Lafontaine, Anne-Louise, Langlois, Mélanie, Leveille, Etienne, Lévesque, Martin, Melmed, Calvin, Monchi, Oury, Montplaisir, Jacques, Panisset, Michel, Parent, Martin, Pham-An, Minh-Thy, Postuma, Ronald, Pourcher, Emmanuelle, Rao, Trisha, Rivest, Jean, Rouleau, Guy, Sharp, Madeleine, Soland, Valérie, Sidel, Michael, Wing Sun, Sonia Lai, Thiel, Alexander, Vitali, Paolo, da Silva Castanheira, Jason, Wiesman, Alex I., Hansen, Justine Y., and Misic, Bratislav

Published
2024
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6. Safety and efficacy of arimoclomol in patients with early amyotrophic lateral sclerosis (ORARIALS-01): a randomised, double-blind, placebo-controlled, multicentre, phase 3 trial

Author

Granit, Volkan, Steele, Julie, Levy, Wendy, Paredes, Maria Elena, Hernandez, Jessica, Bilsker, Martin, Szacka, Katarzyna, Ronert, Adam, Jablońska, Dorota, Łuczak, Alina Zuzanna, Chaverri, Delia, Janse van Mantgem, Mark R, Bunte, Tommy M, Broere, Bianca, de Fockert, Arianne, Sanchez-Tejerina, Daniel, Landabaso, Carmen, Calvo, Andrea, Moglia, Cristina, Manera, Umberto, Canosa, Antonio, Vasta, Rosario, Salamone, Paolina, Fuda, Giuseppe, DeMarco, Giovanni, Casale, Federico, ME Forsberg, Karin, Winroth, Ivar, Almgren Stenberg, Erica, Holmgren, Monica, Amador, Maria del Mar, Lenglet, Timothee, Querin, Giorgia, Coudoin, Sylvie, Pavlakis, Pantelis, Holzberg, Shara, Sideri, Riccardo, Marinou, Kalliopi, Czarnecki, Maciej, Ługiewicz, Renata, Biel-Czarnecka, Marta, Boczkowska, Marcelina, Schotte, Caroline, Vynckier, Jan, Van Daele, Sien, Claeys, Thomas, Delmotte, Koen, Swinnen, Bart, Serrien, Anouk, D'Hondt, Ann, Lamaire, Nikita, Debien, Elisa, Jones, Sarah, Vachon, Chris, Grogan, James, Solorzano, Guillermo, Crowell, Allison, Rakocevic, Goran, Wagoner, Mary, Alma, Osmanovic, Flavia, Wiehler, Sonja, Körner, Olivia, Schreiber-Katz, Camilla, Wohnrade, Anastasia, Sarikidi, Carola, Kassebaum, Chantal, Fischer, Adamo, Ashley, Turcotte, Nicole, Duncan, Jessie, Turner, Ivone, Elman, Lauren, Massie, Rami, Berube, Maxime, Saunders, Natalie, Salmon, Kristiana, Foucher, Juliette, Agessandro, Abrahao, Shirley, Pham, Jahan, Mookshah, Phung, Liane, Statland, Jeffrey, Jawdat, Omar, Dimachkie, Mazen, Pasnoor, Mamatha, Farmakidis, Constantine, Heim, Andrew, Lillig, Katie, Lackey, Alyssa, Weber, Markus, Kurz, Martina, Levine, Todd, Benatar, Michael, Hansen, Thomas, Rom, Dror, Geist, Marie A, Blaettler, Thomas, Camu, William, Kuzma-Kozakiewicz, Magdalena, 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, Elliott, Matthew, Petri, Susanne, Olney, Nicholas, Ladha, Shafeeq, Goyal, Namita A, Meyer, Thomas, Hanna, Michael G, Quinn, Colin, Genge, Angela, 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, and Sundgreen, Claus

Published
2024
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7. Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial

Author

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, and Jiang, John G

Published
2024
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8. Investigating Late-Onset Pompe Prevalence in Neuromuscular Medicine Academic Practices: The IPaNeMA Study.

Author

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, and Mozaffar, Tahseen

Subjects
Genetic Testing, Liver Disease, Digestive Diseases, Chronic Liver Disease and Cirrhosis, Genetics, Rare Diseases, Clinical Research, Neurosciences
Abstract

Background and objectivesWe investigated the prevalence of late-onset Pompe disease (LOPD) in patients presenting to 13 academic, tertiary neuromuscular practices in the United States and Canada.MethodsAll successive patients presenting with proximal muscle weakness or isolated hyperCKemia and/or neck muscle weakness to these 13 centers were invited to participate in the study. Whole blood was tested for acid alpha-glucosidase (GAA) assay through the fluorometric method, and all cases with enzyme levels of ≤10 pmoL/punch/h were reflexed to molecular testing for mutations in the GAA gene. Clinical and demographic information was abstracted from their clinical visit and, along with study data, entered into a purpose-built REDCap database, and analyzed at the University of California, Irvine.ResultsGAA enzyme assay results were available on 906 of the 921 participants who consented for the study. LOPD was confirmed in 9 participants (1% prevalence). Another 9 (1%) were determined to have pseudodeficiency of GAA, whereas 19 (1.9%) were found to be heterozygous for a pathogenic GAA mutation (carriers). Of the definite LOPD participants, 8 (89%) were Caucasian and were heterozygous for the common leaky (IVS1) splice site mutation in the GAA gene (c -32-13T>G), with a second mutation that was previously confirmed to be pathogenic.DiscussionThe prevalence of LOPD in undiagnosed patients meeting the criteria of proximal muscle weakness, high creatine kinase, and/or neck weakness in academic, tertiary neuromuscular practices in the United States and Canada is estimated to be 1%, with an equal prevalence rate of pseudodeficiency alleles.Trial registration informationClinical trial registration number: NCT02838368.

Published
2021

10. Efficacy and safety of iscalimab, a novel anti-CD40 monoclonal antibody, in moderate-to-severe myasthenia gravis: A phase 2 randomized study

Author

GomezMancilla, Baltazar, Meriggioli, Matthew N., Genge, Angela, Roubenoff, Ronenn, Espié, Pascal, Dupuy, Cyrielle, Hartmann, Nicole, Pezous, Nicole, Kinhikar, Arvind, Tichy, Mia, Dionne, Annie, Vissing, John, Andersen, Henning, Schoser, Benedikt, Meisel, Andreas, Jordan, Berit, Devlikamova, Farida, Poverennova, Irina, Stuchevskaya, Fatima, Lin, Thy-Sheng, Rush, James S., and Gergely, Peter

Published
2024
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12. Validating Automatic Diadochokinesis Analysis Methods across Dysarthria Severity and Syllable Task in Amyotrophic Lateral Sclerosis

Author

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, and Yunusova, Yana

Abstract

Purpose: Oral diadochokinesis (DDK) is a standard dysarthria assessment task. To extract automatic and semi-automatic DDK measurements, numerous DDK analysis algorithms based on acoustic signal processing are available, including amplitude based, spectral based, and hybrid. However, these algorithms have been predominantly validated in individuals with no perceptible to mild dysarthria. The behavior of these algorithms across dysarthria severity is largely unknown. Likewise, these algorithms have not been tested equally for various syllable types. The goal of this study was to evaluate the performance of five common DDK algorithms as a function of dysarthria severity, considering syllable types. Method: We analyzed 282 DDK recordings of /ba/, /pa/, and /ta/ from 145 participants with amyotrophic lateral sclerosis. Recordings were stratified into mild, moderate, or severe dysarthria groups based on individual performance on the Speech Intelligibility Test. Analysis included manual and automatic estimation of the number of syllables, DDK rate, and cycle-to-cycle temporal variability (cTV). Validation metrics included Bland-Altman mixed-effects limits of agreement between manual and automatic syllable counts, recall and precision between manual and automatic syllable boundary detection, and Kendall's tau-b correlations between manual and algorithm-detected DDK rate and cTV. Results: The amplitude-based algorithm (absolute energy) yielded the strongest correlations with manual analysis across all severity groups for DDK rate ([tau][subscript b] = 0.7-0.84) and cTV ([tau][subscript b] = 0.7-0.84) and the narrowest limits of agreement (-5.92 to 7.12 syllable difference). Moreover, this algorithm also provided the highest mean recall and precision across severity groups for /ba/ and /pa/, but with significantly more variation for/ta/. Conclusions: Algorithms based on signal energy analysis appeared to be the most robust for DDK analysis across dysarthria severity and syllable types; however, it remains prone to error against severe dysarthria and alveolar syllable context. Further development is needed to address this important issue.

Published
2022
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13. Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study.

Author

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, and Day, John W

Subjects
Muscle, Skeletal, Humans, Muscular Atrophy, Spinal, Troponin I, Drugs, Investigational, Cohort Studies, Double-Blind Method, Adolescent, Adult, Aged, Middle Aged, Child, Female, Male, Young Adult, Walk Test, Reldesemtiv, pharmacodynamics, pharmacokinetics, six-minute walk test, spinal muscular atrophy clinical trial, Clinical Research, Clinical Trials and Supportive Activities, Rare Diseases, 6.1 Pharmaceuticals, Neurosciences, Pharmacology and Pharmaceutical Sciences, Public Health and Health Services, Neurology & Neurosurgery
Abstract

This phase 2, double-blind, placebo-controlled, hypothesis-generating study evaluated the effects of oral reldesemtiv, a fast skeletal muscle troponin activator, in patients with spinal muscular atrophy (SMA). Patients ≥ 12 years of age with type II, III, or IV SMA were randomized into 2 sequential, ascending reldesemtiv dosing cohorts (cohort 1: 150 mg bid or placebo [2:1]; cohort 2: 450 mg bid or placebo [2:1]). The primary objective was to determine potential pharmacodynamic effects of reldesemtiv on 8 outcome measures in SMA, including 6-minute walk distance (6MWD) and maximum expiratory pressure (MEP). Changes from baseline to weeks 4 and 8 were determined. Pharmacokinetics and safety were also evaluated. Patients were randomized to reldesemtiv 150 mg, 450 mg, or placebo (24, 20, and 26, respectively). The change from baseline in 6MWD was greater for reldesemtiv 450 mg than for placebo at weeks 4 and 8 (least squares [LS] mean difference, 35.6 m [p = 0.0037] and 24.9 m [p = 0.058], respectively). Changes from baseline in MEP at week 8 on reldesemtiv 150 and 450 mg were significantly greater than those on placebo (LS mean differences, 11.7 [p = 0.038] and 13.2 cm H2O [p = 0.03], respectively). For 6MWD and MEP, significant changes from placebo were seen in the highest reldesemtiv peak plasma concentration quartile (Cmax > 3.29 μg/mL; LS mean differences, 43.3 m [p = 0.010] and 28.8 cm H2O [p = 0.0002], respectively). Both dose levels of reldesemtiv were well tolerated. Results suggest reldesemtiv may offer clinical benefit and support evaluation in larger SMA patient populations.

Published
2021

14. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study

Author

Genge, Angela, Massie, Rami, Berube, Maxime, Bril, Vera, Daniyal, Lubna, Mannan, Shabber, Ng, Eduardo, Raman, Ritesh Rohan Raghu, Sarpong, Evelyn, Alcantara, Monica, Dionne, Annie, Siddiqi, Zaeem, Blackmore, Derrick, Hussain, Faraz, Matte, Genevieve, Botez, Stephan, Tyblova, Michaela, Jakubikova, Michala, Junkerova, Jana, Vissing, John, Witting, Nanna, Holm-Yildiz, Sonja, Stemmerik, Mads, Andersen, Henning, Obál, Izabella, Solé, Guilhem, Mathis, Stéphane, Violleau, Marie-Hélène, Tranchant, Christine, Messai, Sihame, Chanson, Jean-Baptiste, Nadaj-Pakleza, Aleksandra, Verloes, Arnaud, Zaidi, Leila, Sacconi, Sabrina, Gambella, Manuela, Cavalli, Michele, Stojkovic, Tanya, Demeret, Sophie, Le Guennec, Loic, Querin, Giorgia, Weiss, Nicolas, Masingue, Marion, Magy, Laurent, Ghorab, Karima, Rukhadze, Ia, Tsiskaridze, Alexander, Janelidze, Marina, Margania, Temur, Then Bergh, Florian, Hänsel, Eike, Kalb, Andrea, Meilick, Bianca, Reuschel, Mandy, Teußer, Lars-Malte, Unterlauft, Astrid, Goedel, Clemens, Hagenacker, Tim, Totzeck, Andreas, Stolte, Benjamin, Blaes, Franz, Bindler, Christine, Tsoutsikas, Vasilios, Roediger, Annekathrin, Geis, Christian, Schmidt, Jens, Zschüntzsch, Jana, Schwarz, Margret, Meyer, Stefanie, Kummer, Karsten, Glaubitz, Stefanie, Zeng, Rachel, Wiendl, Heinz, Klotz, Luisa, Lammerskitten, Anna, Lünemann, Jan, Diószeghy, Péter, Mantegazza, Renato, Maggi, Lorenzo, Rinaldi, Elena, Gastaldi, Matteo, Mazzacane, Federico, Businaro, Pietro, Iorio, Raffaele, Antonini, Giovanni, Fionda, Laura, Rinaldi, Rita, Rossi, Simone, Habetswallner, Francesco, Tuccillo, Francesco, Umehara, Haruna, Uenaka, Eiko, Takahashi, Masanori, Higashi, Keiko, Kinoshita, Makoto, Yoneda, Emika, Nakamura, Noriko, Fujita, Saeka, Kubota, Tomoya, Ono, Masami, Yamamoto, Sana, Hatano, Taku, Oikoshi, Kazuki, Yokoyama, Kazumasa, Oji, Yutaka, Tomizawa, Yuji, Uzawa, Akiyuki, Yasuda, Manato, Akita, Sachiko, Ozawa, Yukiko, Onishi, Yosuke, Takaki, Miki, Yamada, Hiromi, Minemoto, Kanako, Sanko, Miki, Izawa, Nanae, Nakayama, Mayumi, Masuda, Masayuki, Tsuji, Rune, Ido, Nobuhiro, Hyodo, Yumi, Okubo, Yoshihiko, Minohara, Akiko, Haraguchi, Nana, Naito, Makiko, Yoshida, Seiko, Fukushige, Yuri, Tsujino, Akira, Nagaoka, Atsushi, Miyazaki, Teiichiro, Yoshimura, Shunsuke, Hirayama, Takuro, Shima, Tomoaki, Okamoto, Naoko, Matsumoto, Riki, Sekiguchi, Kenji, Ueda, Takehiro, Chihara, Norio, Kirimura, Mari, Sunagawa, Emi, Suzuki, Ayaka, Suzuki, Shigeaki, Wada, Aozora, Ishizuchi, Kei, Suzuki, Yasushi, Yata, Mitsuo, Komatsu, Yuka, Tsukita, Kenichi, Watanabe, Genya, Sato, Kazuki, Kawasaki, Emiko, Yamamoto, Naoki, Ono, Hirohiko, Tsuda, Tomoko, Ohashi, Shigeki, Utsugisawa, Kimiaki, Fujisawa, Yuka, Yokota, Yumiko, Nagane, Yuriko, Ayumi, Kameda, Takematsu, Yuka, Naito, Hiroyuki, Sugimoto, Takamichi, Kuwada, Kumiko, Rejdak, Konrad, Szklener, Sebastian, Kitowska, Monika, Derkacz, Kandyda, Druzdz, Artur, Berkowicz, Tomasz, Budzinska, Paulina, Halas, Marek, Zaslavskiy, Leonid, Skornyakova, Evgeniya, Kotov, Sergey, Novikova, Ekaterina, Sidorova, Olga, Goldobin, Vitalii, Alekseeva, Tatiana, Isabekova, Patimat, Malkova, Nadezhda, Korobko, Denis, Djordjevic, Gordana, Stojanov, Aleksandar, Peric, Stojan, Lavrnic, Dragana, Bozovic, Ivo, Palibrk, Aleksa, Casasnovas, Carlos, Nedkova-Hristova, Velina, Vidal Fernández, Nuria, Cortés Vicente, Elena, Querol Gutiérrez, Luis, Salvadó Figueras, Maria, Canovas Segura, Anna, Juntas Morales, Raúl, Sanchez Tejerina, Daniel, Saiz, Albert, Blanco Morgado, Yolanda, Llufriú Durán, Sara, Sepúlveda Gázquez, María, Martínez Hernández, Eugenia María, Gutiérrez Gutiérrez, Gerardo, Iniesta, Paqui, Meca Lallana, José, Guo, Yuh-Cherng, Chiu, Hou-Chang, Yeh, Jiann-Horng, Chen, Ya Hui, Lee, Mei Fen, Lee, Yi-Chung, Lai, Kuan Lin, Beydoun, Said, Akhter, Salma, Vu, Tuan, Lam, Lucy, Thomas, Alisha, Rivner, Michael, Quarles, Brandy, Lange, Dale, Holzberg, Shara, Pavlakis, Pantelis, Goutham, Ashwathy, Kaminski, Henry, Aly, Radwa, Ashworth, Lisa, Bender, Kathryn, Bond, Karie, Buckner, Joanne, Byerly, Sara, Caress, James, Clemons, Jessyca, Farmer, Asha, Franklin, Catherine, Harris, Summer, Hiatt, Meredith, Gandhi Mehta, Rachana, Miller, Gina, Smith, Lynn, Smith, Rose, Strittmatter, Brian, Mozaffar, Tahseen, Habib, Ali A, Hernandez, Isela, Moulton, Kelsey, Karam, Chafic, Ravikumar, Pranali, Lomen-Hoerth, Catherine, Rosow, Laura, George, Hannah, Irodenko, Viktoriya, Kang, Min, Denny, Carol, Hanson, Bart, Klein, Sara, Martinez-Thompson, Jennifer, Naddaf, Elie, Padgett, Denny, Sorenson, Eric, L Sultze, Jane, Weis, Delena, Rezania, Kourosh, Thonhoff, Jason, Shroff, Sheetal, Pascuzzi, Robert, Micheels, Angela, Bodkin, Cynthia, Comer, Adam, Baras, Gelasio, Wagner, Renee, Mahuwala, Zabeen, Ryan, Stephen, Su, Kai, Sharma, Khema, Brown, Andrew, Liow, Kore, Drużdż, Artur, Grosskreutz, Julian, Boehnlein, Marion, Bozorg, Ali, Gayfieva, Maryam, Greve, Bernhard, Woltering, Franz, and Kaminski, Henry J

Published
2023
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15. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study

Author

Sembinelli, Dylan, Teitelbaum, Jeanne, Nicolle, Michael, Bernard, Emilien, Svahn, Juliette, Spinazzi, Marco, Stojkovic, Tanya, Demeret, Sophie, Weiss, Nicolas, Le Guennec, Loïc, Messai, Sihame, Tranchant, Christine, Nadaj-Pakleza, Aleksandra, Chanson, Jean-Baptiste, Suliman, Muhtadi, Zaidi, Leila, Tard, Celine, Lecointe, Peggy, Zschüntzsch, Jana, Schmidt, Jens, Glaubitz, Stefanie, Zeng, Rachel, Scholl, Matthias, Kowarik, Markus, Ziemann, Ulf, Krumbholz, Markus, Martin, Pascal, Ruschil, Christoph, Dünschede, Jutta, Kemmner, Roswitha, Rumpel, Natalie, Berger, Benjamin, Totzeck, Andreas, Hagenacker, Tim, Stolte, Benjamin, Iorio, Raffaele, Evoli, Amelia, Falso, Silvia, Antozzi, Carlo, Frangiamore, Rita, Vanoli, Fiammetta, Rinaldi, Elena, Deguchi, Kazushi, Minami, Naoya, Nagane, Yuriko, Suzuki, Yasushi, Ishida, Sayaka, Suzuki, Shigeaki, Nakahara, Jin, Nagaoka, Astushi, Yoshimura, Shunsuke, Konno, Shingo, Tsuya, Youko, Uzawa, Akiyuki, Kubota, Tomoya, Takahashi, Masanori, Okuno, Tatsusada, Murai, Hiroyuki, Gilhus, Nils Erik, Boldingh, Marion, Rønning, Tone Hakvåg, Chyrchel-Paszkiewicz, Urszula, Kumor, Klaudiusz, Zielinski, Tomasz, Banaszkiewicz, Krzysztof, Błaż, Michał, Kłósek, Agata, Świderek-Matysiak, Mariola, Szczudlik, Andrzej, Paśko, Aneta, Szczechowski, Lech, Banach, Marta, Ilkowski, Jan, Kapetanovic Garcia, Solange, Ortiz Bagan, Patricia, Belén Cánovas Segura, Ana, Turon Sans, Joana, Vidal Fernandez, Nuria, Cortes Vicente, Elena, Rodrigo Armenteros, Patricia, Ashraghi, Mohammad, Cavey, Ana, Haslam, Liam, Emery, Anna, Liow, Kore, Yegiaian, Sharon, Barboi, Alexandru, Vazquez, Rosa Maria, Lennon, Joshua, Pascuzzi, Robert M, Bodkin, Cynthia, Guingrich, Sandra, Comer, Adam, Bromberg, Mark, Janecki, Teresa, Saba, Sami, Tellez, Marco, Elsheikh, Bakri, Freimer, Miriam, Heintzman, Sarah, Govindarajan, Raghav, Guptill, Jeffrey, Massey, Janice M, Juel, Vern, Gonzalez, Natalia, Habib, Ali A, Mozaffar, Tahseen, Korb, Manisha, Goyal, Namita, Machemehl, Hannah, Manousakis, Georgios, Allen, Jeffrey, Harper, Emily, Farmakidis, Constantine, Saavedra, Lilli, Dimachkie, Mazen, Pasnoor, Mamatha, Akhter, Salma, Beydoun, Said, McIlduff, Courtney, Nye, Joan, Roy, Bhaskar, Munro Sheldon, Bailey, Nowak, Richard, Barnes, Benjamin, Rivner, Michael, Suresh, Niraja, Shaw, Jessica, Harvey, Brittany, Lam, Lucy, Thomas, Nikki, Chopra, Manisha, Traub, Rebecca E, Jones, Sarah, Wagoner, Mary, Smajic, Sejla, Aly, Radwa, Katz, Jonathan, Chen, Henry, Miller, Robert G, Jenkins, Liberty, Khan, Shaida, Khatri, Bhupendra, Sershon, Lisa, Pavlakis, Pantelis, Holzberg, Shara, Li, Yuebing, Caristo, Irys B, Marquardt, Robert, Hastings, Debbie, Rube, Jacob, Lisak, Robert P, Choudhury, Aparna, Ruzhansky, Katherine, Sachdev, Amit, Shin, Susan, Bratton, Joan, Fetter, Mary, McKinnon, Naya, McKinnon, Jonathan, Sissons-Ross, Laura, Sahu, Amos, Distad, B Jane, Howard, James F, Jr, Bresch, Saskia, Genge, Angela, Hewamadduma, Channa, Hinton, John, Hussain, Yessar, Juntas-Morales, Raul, Kaminski, Henry J, Maniaol, Angelina, Mantegazza, Renato, Masuda, Masayuki, Sivakumar, Kumaraswamy, Śmiłowski, Marek, Utsugisawa, Kimiaki, Vu, Tuan, Weiss, Michael D, Zajda, Małgorzata, Boroojerdi, Babak, Brock, Melissa, de la Borderie, Guillemette, Duda, Petra W, Lowcock, Romana, Vanderkelen, Mark, and Leite, M Isabel

Published
2023
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16. Addressing heterogeneity in amyotrophic lateral sclerosis CLINICAL TRIALS

Author

Goyal, Namita A, Berry, James D, Windebank, Anthony, Staff, Nathan P, Maragakis, Nicholas J, Berg, Leonard H, Genge, Angela, Miller, Robert, Baloh, Robert H, Kern, Ralph, Gothelf, Yael, Lebovits, Chaim, and Cudkowicz, Merit

Subjects
Biological Sciences, Biomedical and Clinical Sciences, Genetics, Neurosciences, Orphan Drug, Clinical Research, Rare Diseases, ALS, Brain Disorders, Neurodegenerative, Clinical Trials and Supportive Activities, Detection, screening and diagnosis, 4.1 Discovery and preclinical testing of markers and technologies, Neurological, Good Health and Well Being, Amyotrophic Lateral Sclerosis, Biological Variation, Population, Biomarkers, Clinical Trials as Topic, Disease Progression, Drug Development, Humans, Muscle Strength, Outcome Assessment, Health Care, Physical Functional Performance, Precision Medicine, Prognosis, Reproducibility of Results, Respiratory Function Tests, Risk Assessment, Speech, Transcranial Magnetic Stimulation, amyotrophic lateral sclerosis, biomarkers, clinical trials, disease heterogeneity, enrichment strategies, outcome measures, Medical and Health Sciences, Neurology & Neurosurgery, Biological sciences, Biomedical and clinical sciences
Abstract

Amyotrophic lateral sclerosis (ALS) is a debilitating neurodegenerative disorder with complex biology and significant clinical heterogeneity. Many preclinical and early phase ALS clinical trials have yielded promising results that could not be replicated in larger phase 3 confirmatory trials. One reason for the lack of reproducibility may be ALS biological and clinical heterogeneity. Therefore, in this review, we explore sources of ALS heterogeneity that may reduce statistical power to evaluate efficacy in ALS trials. We also review efforts to manage clinical heterogeneity, including use of validated disease outcome measures, predictive biomarkers of disease progression, and individual clinical risk stratification. We propose that personalized prognostic models with use of predictive biomarkers may identify patients with ALS for whom a specific therapeutic strategy may be expected to be more successful. Finally, the rapid application of emerging clinical and biomarker strategies may reduce heterogeneity, increase trial efficiency, and, in turn, accelerate ALS drug development.

Published
2020

19. Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials.

Author

van den Berg, Leonard, Sorenson, Eric, Gronseth, Gary, Macklin, Eric, Andrews, Jinsy, Baloh, Robert, Benatar, Michael, Berry, James, Chio, Adriano, Corcia, Philippe, Genge, Angela, Gubitz, Amelie, Lomen-Hoerth, Catherine, McDermott, Christopher, Pioro, Erik, Rosenfeld, Jeffrey, Silani, Vincenzo, Turner, Martin, Weber, Markus, Brooks, Benjamin, Miller, Robert, and Mitsumoto, Hiroshi

Subjects
Amyotrophic Lateral Sclerosis, Biomarkers, Clinical Trials as Topic, Delphi Technique, Guidelines as Topic, Humans, Outcome Assessment, Health Care, Patient Selection, Research Design, Statistics as Topic
Abstract

OBJECTIVE: To revise the 1999 Airlie House consensus guidelines for the design and implementation of preclinical therapeutic studies and clinical trials in amyotrophic lateral sclerosis (ALS). METHODS: A consensus committee comprising 140 key members of the international ALS community (ALS researchers, clinicians, patient representatives, research funding representatives, industry, and regulatory agencies) addressed 9 areas of need within ALS research: (1) preclinical studies; (2) biological and phenotypic heterogeneity; (3) outcome measures; (4) disease-modifying and symptomatic interventions; (5) recruitment and retention; (6) biomarkers; (7) clinical trial phases; (8) beyond traditional trial designs; and (9) statistical considerations. Assigned to 1 of 8 sections, committee members generated a draft set of guidelines based on a background of developing a (pre)clinical question and a rationale outlining the evidence and expert opinion. Following a 2-day, face-to-face workshop at the Airlie House Conference Center, a modified Delphi process was used to develop draft consensus research guidelines, which were subsequently reviewed and modified based on comments from the public. Statistical experts drafted a separate document of statistical considerations (section 9). RESULTS: In this report, we summarize 112 guidelines and their associated backgrounds and rationales. The full list of guidelines, the statistical considerations, and a glossary of terms can be found in data available from Dryad (appendices e-3-e-5, doi.org/10.5061/dryad.32q9q5d). The authors prioritized 15 guidelines with the greatest potential to improve ALS clinical research. CONCLUSION: The revised Airlie House ALS Clinical Trials Consensus Guidelines should serve to improve clinical trial design and accelerate the development of effective treatments for patients with ALS.

Published
2019

21. The C-BIG Repository: an Institution-Level Open Science Platform

Author

Das, Samir, Abou-Haidar, Rida, Rabalais, Henri, Sun, Sonia Denise Lai Wing, Rosli, Zaliqa, Chatpar, Krishna, Boivin, Marie-Noëlle, Tabatabaei, Mahdieh, Rogers, Christine, Legault, Melanie, Lo, Derek, Degroot, Clotilde, Dagher, Alain, Dyke, Stephanie O. M., Durcan, Thomas M., Seyller, Annabel, Doyon, Julien, Poupon, Viviane, Fon, Edward A., Genge, Angela, Rouleau, Guy A., Karamchandani, Jason, and Evans, Alan C.

Published
2022
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22. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

Author

Behin, Anthony, Boentert, Matthias, Carvalho, Gerson, Chahin, Nizar, Charrow, Joel, Deegan, Patrick, Durmus Tekce, Hacer, Duval, Fanny, Genge, Angela, Gutmann, Ludwig, Henderson, Robert D, Hennermann, Julia B, Hiwot, Tarekegn, Hughes, Derralynn, Karaa, Amel, Karam, Chafic, Kautzky-Willer, Alexandra, Komaki, Hirofumi, Laforet, Pascal, Longo, Nicola, Malinova, Vera, Maré, Ricardo, Maxit, Clarisa, Mengel, Eugen, Moggio, Maurizio Gualtiero, Molnár, Mária Judit, Mongini, Tiziana Enrica, Nadaj-Pakleza, Aleksandra, Nascimento Osorio, Andres, Noury, Jean-Baptiste, Oliveira, Acary Souza Bulle, Parman, Yesim, Pena, Loren, Remiche, Gauthier, Sciacco, Monica, Shieh, Perry B, Smith, Cheryl, Stulnig, Thomas, Taithe, Frederic, Tard, Céline, Tarnopolsky, Mark, Vorgerd, Matthias, Whitley, Chester, Young, Peter, Alonso-Pérez, Jorge, Altemus, Patricia, Aubé-Nathier, Anne-Catherine, Avelar, Jennifer B, Bailey, Carrie, Bekircan-Kurt, Can Ebru, Billy, Jenny, Boschi, Silvia, Brown, Kathryn E, Carrera Garcia, Laura, Chase, Lauren, Cirne, Hamilton, Danjoux, Loïc, Davion, Jean-Baptiste, DeArmey, Stephanie, Fedotova, Ekaterina, Gandolfo, Eve, Grosz, Zoltan, Guellec, Dewi, Guettsches, Anne-Katrin, Guglieri, Michela, Hatcher, Erin, Helms, Sina, Hufgard-Leitner, Miriam, Klyushnikov, Sergey A., Langton, Jacqui, Linková, Lenka, Mavroudakis, Nicolas, Mazurová, Stella, Mori, Madoka, Müller-Miny, Louisa, Musumeci, Olimpia, Nance, Christopher S, Natera-de Benito, Daniel, Neel, Robert, Niizawa, Gabriela A, Noll, Lauren, Ortega, Erik, Pasnoor, Mamatha, Pautot, Vivien, Potulska-Chromik, Anna, Pugliese, Alessia, Questienne, Claire, Ramos Lopes, Margarida, Reyes-Leiva, David, Riedl, Michaela, Rugiero, Marcelo Francisco, Salort-Campana, Emmanuelle, Sgobbi Souza, Paulo Victor, Sole, Guilhem, Solera, Luca, Souto Lopes, Suzara, Specht, Sabine, Statland, Jeffrey, Swenson, Andrea, Tan, Chong Yew, Tizon, Sónia, van der Beek, N A M E, van Kooten, Harmke A., Wencel, Marie, Wenninger, Stephan, Zagnoli, Fabien, Diaz-Manera, Jordi, Kishnani, Priya S, Kushlaf, Hani, Ladha, Shafeeq, Mozaffar, Tahseen, Straub, Volker, Toscano, Antonio, van der Ploeg, Ans T, Berger, Kenneth I, Clemens, Paula R, Chien, Yin-Hsiu, Day, John W, Illarioshkin, Sergey, Roberts, Mark, Attarian, Shahram, Borges, Joao Lindolfo, Bouhour, Francoise, Choi, Young Chul, Erdem-Ozdamar, Sevim, Goker-Alpan, Ozlem, Kostera-Pruszczyk, Anna, Haack, Kristina An, Hug, Christopher, Huynh-Ba, Olivier, Johnson, Judith, Thibault, Nathan, Zhou, Tianyue, Dimachkie, Mazen M, and Schoser, Benedikt

Published
2021
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24. QRL-201-01—A Multi-center, Randomized, Double-blind, Placebo-controlled Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of QRL-201 in Amyotrophic Lateral Sclerosis (P6-11.010)

Author

Genge, Angela, primary, Salmon, Kristiana, additional, Polzer, John, additional, Martinez, Clarida, additional, Boggs, Bryan, additional, Eon, Victoria, additional, Ganti, Rakhee, additional, Hinckley, Sandy, additional, Johnson, Kristina, additional, and Elbaum, Daniel, additional

Published
2024
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27. Long-term Safety and Efficacy of Zilucoplan in Myasthenia Gravis: Additional Interim Analyses of RAISE-XT (S15.002)

Author

Howard, James F., primary, Freimer, Miriam, additional, Genge, Angela, additional, Hewamadduma, Channa, additional, Hussain, Yessar, additional, Maniaol, Angelina, additional, Mantegazza, Renato, additional, Smilowski, Marek, additional, Utsugisawa, Kimiaki, additional, Vu, Tuan, additional, Weiss, Michael, additional, Duda, Petra, additional, Boroojerdi, Babak, additional, Vanderkelen, Mark, additional, De La Borderie, Guillemette, additional, and Leite, M. Isabel, additional

Published
2024
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28. Safety and efficacy of oral levosimendan in people with amyotrophic lateral sclerosis (the REFALS study): a randomised, double-blind, placebo-controlled phase 3 trial

Author

Kiernan, Matthew, Mathers, Susan, Henderson, Robert, Needham, Merrilee, Schultz, David, Löscher, Wolfgang, Mitrovic, Nenad, Rath, Jakob, Damme, Philip Van, De Bleecker, Jan L., Delstanche, Stéphanie, Johnston, Wendy, Zinman, Lorne, O'Connell, Colleen, Matte, Genevieve, Dionne, Annie, Korngut, Lawrence, Turnbull, John, Laaksovirta, Hannu, Jokela, Manu, Tapiola, Tero, Soriani, Marie-Hélène, Couratier, Philippe, Camu, William, Corcia, Philippe, Ludolph, Albert, Großkreutz, Julian, Meyer, Thomas, Boentert, Matthias, Schrank, Berthold, Prudlo, Johannes, Untucht, Robert, Hardiman, Orla, Siciliano, Gabriele, Chio', Adriano, Mazzini, Letizia, Inghilleri, Maurizio, Caponnetto, Claudia, Mora, Gabriele, Mora Pardina, Jesús S, Farrero Munoz, Eva, Vázquez Costa, Juan F, Aguera Morales, Eduardo, Varona, Luis, Andersen, Peter, Ingre, Caroline, Johansson, Rune, Radunovic, Aleksandar, Young, Carolyn, Babu, Suma, Shaibani, Aziz, Staff, Nathan, Vu, Tuan, Rivner, Michael, Scelsa, Stephen, Sivakumar, Kumaraswamy, Waheed, Waqar, Heitzman, Daragh, Rana, Sandeep, Pattee, Gary, Ajroud-Driss, Senda, Bayat, Elham, Kasarskis, Edward, Lange, Dale J, Elliott, Michael, Harris, Brent, Felice, Kevin, Pulley, Michael T, Kwan, Justin, Brown, Martin, Ravits, John, Burford, Matthew, Karam, Chafic, Miller, Timothy, Andrews, Jinsy, Levine, Todd, Locatelli, Eduardo, Wymer, James, Bedlack, Richard, Fee, Dominic, Goyal, Namita, Oskarsson, Bjorn, McCluskey, Leo, Caress, James, Weiss, Michael, Quick, Adam, Bromberg, Mark, Lacomis, David, Goutman, Stephen, Rezania, Kourosh, Guliani, Gaurav, Goslin, Kimberly, Katz, Jonathan S, Cudkowicz, Merit, Genge, Angela, Maragakis, Nicholas, Petri, Susanne, van den Berg, Leonard, Aho, Valtteri V, Sarapohja, Toni, Kuoppamäki, Mikko, Garratt, Chris, and Al-Chalabi, Ammar

Published
2021
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29. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial

Author

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

Published
2021
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31. Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study

Author

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, and Day, John W.

Published
2021
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32. The ALSFRS-R Summit: a global call to action on the use of the ALSFRS-R in ALS clinical trials.

Author

Genge, Angela, Cedarbaum, Jesse M., Shefner, Jeremy, Chio, Adriano, Al-Chalabi, Ammar, Van Damme, Philip, McDermott, Chris, Glass, Jonathan, Berry, James, van Eijk, Ruben P.A., Fournier, Christina, Grosskreutz, Julian, Andrews, Jinsy, Bertone, Vanessa, Bunte, Tommy M, Couillard, Mathias, Cummings, Cathy, Kittle, Gale, Polzer, John, and Salmon, Kristiana

Subjects
*CLINICAL trials, *AMYOTROPHIC lateral sclerosis, *EXPERIMENTAL design
Abstract

The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) was developed more than 25 years ago as an instrument to monitor functional change over time in patients with ALS. It has since been revised and extended to meet the needs of high data quality in ALS trials (ALSFRS-R), however a full re-validation of the scale was not completed. Despite this, the scale has remained a primary outcome measure in clinical trials. We convened a group of clinical trialists to discuss and explore opportunities to improve the scale and propose alternative measures. In this meeting report, we present a call to action on the use of the ALSFRS-Revised scale in clinical trials, focusing on the need for (1) harmonization of the ALSFRS-R administration globally, (2) alignment on a set of recommendations for clinical trial design and statistical analysis plans (SAPs), and (3) use of additional outcome measures. [ABSTRACT FROM AUTHOR]

Published
2024
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33. Amyotrophic lateral sclerosis: exploring pathophysiology in the context of treatment.

Author

Genge, Angela, Wainwright, Steven, and Vande Velde, Christine

Subjects
*AMYOTROPHIC lateral sclerosis, *PATHOLOGICAL physiology, *THERAPEUTICS, *DRUG development, *NEURODEGENERATION
Abstract

Amyotrophic lateral sclerosis (ALS) is a complex, neurodegenerative disorder in which alterations in structural, physiological, and metabolic parameters act synergistically. Over the last decade there has been a considerable focus on developing drugs to slow the progression of the disease. Despite this, only four disease-modifying therapies are approved in North America. Although additional research is required for a thorough understanding of ALS, we have accumulated a large amount of knowledge that could be better integrated into future clinical trials to accelerate drug development and provide patients with improved treatment options. It is likely that future, successful ALS treatments will take a multi-pronged therapeutic approach, targeting different pathways, akin to personalized medicine in oncology. In this review, we discuss the link between ALS pathophysiology and treatments, looking at the therapeutic failures as learning opportunities that can help us refine and optimize drug development. [ABSTRACT FROM AUTHOR]

Published
2024
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39. Long-term safety, efficacy & self-injection satisfaction with zilucoplan in myasthenia gravis: RAISE-XT interim analysis

Author

Genge, Angela, primary, Leite, M. Isabel, additional, Bresch, Saskia, additional, Freimer, Miriam, additional, Hewamadduma, Channa, additional, Hussain, Yessar, additional, Maniaol, Angelina, additional, Mantegazza, Renato, additional, Smilowski, Marek, additional, Utsugisawa, Kimiaki, additional, Vu, Tuan, additional, Duda, Petra W., additional, Boroojerdi, Babak, additional, Vanderkelen, Mark, additional, De La Borderie, Guillemette, additional, Bloemers, Jos, additional, and Howard, James F., additional

Published
2023
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40. QRL-201-01: A multi-center, randomized, double-blind, placebo-controlled multiple ascending dose study to evaluate the safety and tolerability of QRL-201 in amyotrophic lateral sclerosis

Author

Genge, Angela, primary, Salmon, Kristiana, additional, Polzer, John, additional, Martínez, Clárida, additional, Boggs, Bryan, additional, Eon, Victoria, additional, Ganti, Rakhee, additional, Hinckley, Sandy, additional, Johnson, Kristina, additional, and Elbaum, Daniel, additional

Published
2023
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42. Long-term safety and efficacy of zilucoplan in patients with generalized myasthenia gravis: interim analysis of the RAISE-XT open-label extension study.

Author

Howard Jr, James F., Bresch, Saskia, Farmakidis, Constantine, Freimer, Miriam, Genge, Angela, Hewamadduma, Channa, Hinton, John, Hussain, Yessar, Juntas-Morales, Raul, Kaminski, Henry J., Maniaol, Angelina, Mantegazza, Renato, Masuda, Masayuki, Nowak, Richard J., Sivakumar, Kumaraswamy, Śmiłowski, Marek, Utsugisawa, Kimiaki, Vu, Tuan, Weiss, Michael D., and Zajda, Małgorzata

Subjects
MYASTHENIA gravis, DRUG efficacy, CHOLINERGIC receptors, CLINICAL trials, AUTOANTIBODIES
Abstract

Background: Generalized myasthenia gravis (gMG) is a chronic, unpredictable disease associated with high treatment and disease burdens, with a need for more effective and well-tolerated treatments. Objectives: To evaluate the long-term safety, tolerability, and efficacy of zilucoplan in a mild-to-severe, acetylcholine receptor autoantibody-positive (AChR+) gMG population. Design: Ongoing, multicenter, phase III open-label extension (OLE) study. Methods: Eligible patients had completed a qualifying randomized, placebo-controlled phase II or phase III zilucoplan study and received daily, self-administered subcutaneous 0.3 mg/kg zilucoplan. The primary endpoint was incidence of treatment-emergent adverse events (TEAEs). Secondary efficacy endpoints included change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score. Results: In total, 200 patients enrolled. At the cut-off date (8 September 2022), median (range) exposure to zilucoplan in RAISE-XT was 1.2 (0.11–4.45) years. Mean age at OLE baseline was 53.3 years. A total of 188 (94%) patients experienced a TEAE, with the most common being MG worsening (n = 52, 26%) and COVID-19 (n = 49, 25%). In patients who received zilucoplan 0.3 mg/kg in the parent study, further improvements in MG-ADL score continued through to Week 24 (least squares mean change [95% confidence interval] from double-blind baseline −6.06 [−7.09, −5.03]) and were sustained through to Week 60 (−6.04 [−7.21, −4.87]). In patients who switched from placebo in the parent study, rapid improvements in MG-ADL score were observed at the first week after switching to zilucoplan; further improvements were observed at Week 24, 12 weeks after switching (−6.46 [−8.19, −4.72]), and were sustained through to Week 60 (−6.51 [−8.37, −4.65]). Consistent results were observed in other efficacy endpoints. Conclusion: Zilucoplan demonstrated a favorable long-term safety profile, good tolerability, and sustained efficacy through to Week 60 with consistent benefits in a broad AChR+ gMG population. Additional long-term data will be available in future analyses. Trial registration: ClinicalTrials.gov identifier: NCT04225871 (https://clinicaltrials.gov/ct2/show/NCT04225871) [ABSTRACT FROM AUTHOR]

Published
2024
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43. Speech timing and monosyllabic diadochokinesis measures in the assessment of amyotrophic lateral sclerosis in Canadian French.

Author

Bouvier, Liziane, McKinlay, Scotia, Truong, Justin, Genge, Angela, Dupré, Nicolas, Dionne, Annie, Kalra, Sanjay, and Yunusova, Yana

Subjects
STATISTICAL correlation, PROGRESSIVE bulbar palsy, SECONDARY analysis, DYSARTHRIA, DATA analysis, RESEARCH funding, TEACHING aids, KRUSKAL-Wallis Test, SEVERITY of illness index, DESCRIPTIVE statistics, AGE distribution, AMYOTROPHIC lateral sclerosis, PHYSIOLOGICAL aspects of speech, SPEECH evaluation, RESEARCH, STATISTICS, CONTINUING education, FRENCH-Canadians, COMPARATIVE studies, EARLY diagnosis, SPEECH disorders, EVALUATION, SYMPTOMS
Abstract

The primary objective of this study was to determine if speech and pause measures obtained using a passage reading task and timing measures from a monosyllabic diadochokinesis (DDK) task differ across speakers of Canadian French diagnosed with amyotrophic lateral sclerosis (ALS) presenting with and without bulbar symptoms, and healthy controls. The secondary objective was to determine if these measures can reflect the severity of bulbar symptoms. A total of 29 Canadian French speakers with ALS (classified as bulbar symptomatic [n = 14] or pre-symptomatic [n = 15]) and 17 age-matched healthy controls completed a passage reading task and a monosyllabic DDK task (/pa/ and /ta/), for up to three follow-up visits. Measures of speaking rate, total duration, speech duration, and pause events were extracted from the passage reading recordings using a semi-automated speech and pause analysis procedure. Manual analysis of DDK recordings provided measures of DDK rate and variability. Group comparisons revealed significant differences (p = <.05) between the symptomatic group and the pre-symptomatic and control groups for all passage measures and DDK rates. Only the DDK rate in /ta/ differentiated the pre-symptomatic and control groups. Repeated measures correlations revealed moderate correlations (rrm = > 0.40; p = < 0.05) between passage measures of total duration, speaking rate, speech duration, and number of pauses, and ALSFRS-R total and bulbar scores, as well as between DDK rate and ALSFRS-R total score. Speech and pause measures in passage and timing measures in monosyllabic DDK tasks might be suitable for monitoring bulbar functional symptoms in French speakers with ALS, but more work is required to identify which measures are sensitive to the earliest stages of the disease. [ABSTRACT FROM AUTHOR]

Published
2024
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44. Harmonized standard operating procedures for administering the ALS functional rating scale-revised.

Author

Shefner, Jeremy M., Bunte, Tommy, Kittle, Gale, Genge, Angela, and van den Berg, Leonard H

Subjects
STANDARD operating procedure, CLINICAL trials
Abstract

The ALS Functional Rating Scale-Revised is the most commonly used primary outcome measure in current ALS clinical trials. While rigorous training and certification is generally recognized as critical to reliable performance, differences have existed between training in the two groups responsible for most training in ALS outcome measures. We present a harmonized standard operating procedure which is intended to further reduce response variability by the use of identical training in North America and Europe. [ABSTRACT FROM AUTHOR]

Published
2024
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47. SF2Former: Amyotrophic lateral sclerosis identification from multi-center MRI data using spatial and frequency fusion transformer

Author

Kushol, Rafsanjany, primary, Luk, Collin C., additional, Dey, Avyarthana, additional, Benatar, Michael, additional, Briemberg, Hannah, additional, Dionne, Annie, additional, Dupré, Nicolas, additional, Frayne, Richard, additional, Genge, Angela, additional, Gibson, Summer, additional, Graham, Simon J., additional, Korngut, Lawrence, additional, Seres, Peter, additional, Welsh, Robert C., additional, Wilman, Alan H., additional, Zinman, Lorne, additional, Kalra, Sanjay, additional, and Yang, Yee-Hong, additional

Published
2023
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49. 2603 Safety and tolerability of zilucoplan in RAISE-XT: a multicenter, open-label extension study in patients with myasthenia gravis

Author

Genge, Angela, primary, Hussain, Yessar, additional, Kaminski, Henry J, additional, Isabel Leite, M, additional, Mantegazza, Renato, additional, Utsugisawa, Kimiaki, additional, Vu, Tuan, additional, Brock, Melissa, additional, Boroojerdi, Babak, additional, Vanderkelen, Mark, additional, Borderie, Guillemette de la, additional, Duda, Petra W, additional, Moody, Anita, additional, and Howard, James F, additional

Published
2023
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50. Oral fingolimod for chronic inflammatory demyelinating polyradiculoneuropathy (FORCIDP Trial): a double-blind, multicentre, randomised controlled trial

Author

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, III, Brown, Martin, Kelemen, John, Iyadurai, Stanley, Rezania, Kourosh, Sharma, Khema R, Tandan, Rup, Gudesblatt, Mark, Lawson, Victoria, Amato, Anthony A, 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, and Hartung, Hans-Peter

Published
2018
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