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

Autor: 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 ^{a, b, c, *, *}, Kishnani, Priya S ^{d, *}, Kushlaf, Hani ^e, Ladha, Shafeeq ^f, Mozaffar, Tahseen ^g, Straub, Volker ^a, Toscano, Antonio ^h, van der Ploeg, Ans T ⁱ, Berger, Kenneth I ^{j, k}, Clemens, Paula R ^{l, m}, Chien, Yin-Hsiu ⁿ, Day, John W ^o, Illarioshkin, Sergey ^p, Roberts, Mark ^q, Attarian, Shahram ^r, Borges, Joao Lindolfo ^s, Bouhour, Francoise ^t, Choi, Young Chul ^u, Erdem-Ozdamar, Sevim ^v, Goker-Alpan, Ozlem ^w, Kostera-Pruszczyk, Anna ^x, Haack, Kristina An ^y, Hug, Christopher ^z, Huynh-Ba, Olivier ^aa, Johnson, Judith ^z, Thibault, Nathan ^z, Zhou, Tianyue ^z, Dimachkie, Mazen M ^{ab, †}, Schoser, Benedikt ^{ac, †}

Publikováno v: In The Lancet Neurology December 2021 20(12):1012-1026

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P376 COMET post hoc analysis: efficacy of long-term avalglucosidase alfa in subgroups of patients with late-onset Pompe disease

Autor: Toscano, A. ¹, Kishnani, P. ², Dimachkie, M. ³, Sacconi, S. ⁴, van der Beek, N. ⁵, Roberts, M. ⁶, Suwazono, S. ⁷, Choi, Y. ⁸, de Souza, P Sgobbi ⁹, Schoser, B. ¹⁰, Armstrong, N. ¹¹, Huynh-Ba, O. ¹², Thibault, N. ¹¹, Periquet, M. ¹³, Díaz-Manera, J. ¹⁴

Publikováno v: In Neuromuscular Disorders October 2023 33 Supplement 1:S152-S152