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Kurzansicht	E-Jahr	Zugriff	Typ	Operations
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease 2024 . Kishnani, Priya S. \| Byrne, Barry J. \| Claeys, Kristl \| Díaz-Manera, Jordi \| Dimachkie, Mazen M. \| Kushlaf, Hani \| Mozaffar, Tahseen \| Roberts, Mark \| Schoser, Benedikt \| Hummel, Noemi \| Kopiec, Agnieszka \| Holdbrook, Fred \| Shohet, Simon \| Toscano, Antonio \| on behalf of the PROPEL Study Group \| Sebok, Agnes \| Pestronk, Alan \| Dominovic-Kovacevic, Aleksandra \| Khan, Aneal \| Koritnik, Blaž \| Tard, Celine \| Lindberg, Christopher \| Quinn, Colin \| Eldridge, Crystal \| Bodkin, Cynthia \| Reyes-Leiva, David \| Hughes, Derralynn \| Stefanescu, Ela \| Salort-Campana, Emmanuelle \| Butler, Ernest \| Bouhour, Francoise \| Kim, Gee \| Papadimas, George Konstantinos \| Parenti, Giancarlo \| Bartosik-Psujek, Halina \| Akihiro, Hashiguchi \| Lau, Heather \| Pedro, Helio \| Andersen, Henning \| Amartino, Hernan \| Shiraishi, Hideaki \| Kobayashi, Hiroshi \| Tarnev, Ivaylo \| Vengoechea, Jaime \| Avelar, Jennifer \| Shin, Jin-Hong \| Cauci, Jonathan \| Alonso-Pérez, Jorge \| Janszky, Jozsef \| Berthy, Julie \| Kornblum, Cornelia \| Gutschmidt, Kristina \| Molnar, Maria Judit \| Wencel, Marie \| Tarnopolsky, Mark \| Tchan, Michel \| Freimer, Miriam \| Longo, Nicola \| Vidal-Fernandez, Nuria \| Musumeci, Olimpia \| Goker-Alpan, Ozlem \| Deegan, Patrick \| Clemens, Paula R \| Roxburgh, Richard \| Henderson, Robert \| Hopkin, Robert \| Sacconi, Sabrina \| Fecarotta, Simona \| Attarian, Shahram \| Wenninger, Stephan \| Dearmey, Stephanie \| Hiwot, Tarekegn \| Burrow, Thomas \| Ruck, Tobias \| Sawada, Tomo \| Laszlo, Vescei \| Löscher, Wolfgang \| Chien, Yin-Hsiu	2024		http://purl.org/ontology/bibo/Article
Correction to: Analysis of muscle magnetic resonance imaging of a large cohort of patient with VCP‑mediated disease reveals characteristic features useful for diagnosis 2024 . Esteller, Diana \| Schiava, Marianela \| Verdú-Díaz, José \| Villar-Quiles, Rocío-Nur \| Dibowski, Boris \| Venturelli, Nadia \| Laforet, Pascal \| Alonso-Pérez, Jorge \| Olive, Montse \| Domínguez-González, Cristina \| Paradas, Carmen \| Vélez, Beatriz \| Kostera-Pruszczyk, Anna \| Kierdaszuk, Biruta \| Rodolico, Carmelo \| Claeys, Kristl \| Pál, Endre \| Malfatti, Edoardo \| Souvannanorath, Sarah \| Alonso-Jiménez, Alicia \| de Ridder, Willem \| De Smet, Eline \| Papadimas, George \| Papadopoulos, Constantinos \| Xirou, Sofia \| Luo, Sushan \| Muelas, Nuria \| Vilchez, Juan J. \| Ramos-Fransi, Alba \| Monforte, Mauro \| Tasca, Giorgio \| Udd, Bjarne \| Palmio, Johanna \| Sri, Srtuhi \| Krause, Sabine \| Schoser, Benedikt \| Fernández-Torrón, Roberto \| López de Munain, Adolfo \| Pegoraro, Elena \| Farrugia, Maria Elena \| Vorgerd, Mathias \| Manousakis, Georgious \| Chanson, Jean Baptiste \| Nadaj-Pakleza, Aleksandra \| Cetin, Hakan \| Badrising, Umesh \| Warman-Chardon, Jodi \| Bevilacqua, Jorge \| Earle, Nicholas \| Campero, Mario \| Díaz, Jorge \| Ikenaga, Chiseko \| Lloyd, Thomas E. \| Nishino, Ichizo \| Nishimori, Yukako \| Saito, Yoshihiko \| Oya, Yasushi \| Takahashi, Yoshiaki \| Nishikawa, Atsuko \| Sasaki, Ryo \| Marini-Bettolo, Chiara \| Guglieri, Michela \| Straub, Volker \| Stojkovic, Tanya \| Carlier, Robert Y. \| Diaz-Manera, Jordi	2024		http://purl.org/ontology/bibo/Article
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07) 2024 . Schoser, Benedikt \| Kishnani, Priya S. \| Bratkovic, Drago \| Byrne, Barry J. \| Claeys, Kristl G. \| Díaz-Manera, Jordi \| Laforêt, Pascal \| Roberts, Mark \| Toscano, Antonio \| van der Ploeg, Ans T. \| Castelli, Jeff \| Goldman, Mitchell \| Holdbrook, Fred \| Sitaraman Das, Sheela \| Wasfi, Yasmine \| Mozaffar, Tahseen \| the ATB200-07 Study Group \| Sebok, Agnes \| Pestronk, Alan \| Dominovic-Kovacevic, Aleksandra \| Khan, Aneal \| Koritnik, Blaž \| Tard, Celine \| Lindberg, Christopher \| Quinn, Colin \| Eldridge, Crystal \| Bodkin, Cynthia \| Reyes-Leiva, David \| Hughes, Derralynn \| Stefanescu, Ela \| Salort-Campana, Emmanuelle \| Butler, Ernest \| Bouhour, Francoise \| Kim, Gee \| Papadimas, George Konstantinos \| Parenti, Giancarlo \| Bartosik-Psujek, Halina \| Kushlaf, Hani \| Akihiro, Hashiguchi \| Lau, Heather \| Pedro, Helio \| Andersen, Henning \| Amartino, Hernan \| Shiraishi, Hideaki \| Kobayashi, Hiroshi \| Tarnev, Ivaylo \| Vengoechea, Jaime \| Avelar, Jennifer \| Shin, Jin-Hong \| Nevin, John \| Cauci, Jonathan \| Alonso-Pérez, Jorge \| Janszky, Jozsef \| Berthy, Julie \| Kornblum, Cornelia \| Gutschmidt, Kristina \| Molnar, Maria Judit \| Wencel, Marie \| Tarnopolsky, Mark \| Boentert, Matthias \| Tchan, Michel \| Freimer, Miriam \| Longo, Nicola \| Abreu, Nicolas \| Vidal-Fernandez, Nuria \| Musumeci, Olimpia \| Goker-Alpan, Ozlem \| Deegan, Patrick \| Clemens, Paula R. \| Roxburgh, Richard \| Henderson, Robert \| Hopkin, Robert \| Sacconi, Sabrina \| Fecarotta, Simona \| Attarian, Shahram \| Wenninger, Stephan \| Dearmey, Stephanie \| Hiwot, Tarekegn \| Burrow, Thomas \| Ruck, Tobias \| Sawada, Tomo \| Laszlo, Vescei \| Löscher, Wolfgang \| Chien, Yin-Hsiu	2024		http://purl.org/ontology/bibo/Article
STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa 2021 . Gutschmidt, Kristina \| Musumeci, Olimpia \| Díaz-Manera, Jordi \| Chien, Yin-Hsiu \| Knop, Karl Christian \| Wenninger, Stephan \| Montagnese, Federica \| Pugliese, Alessia \| Tavilla, Graziana \| Alonso-Pérez, Jorge \| Hwu, Paul Wuh-Liang \| Toscano, Antonio \| Schoser, Benedikt	2021		http://purl.org/ontology/bibo/Article