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2 Treffer

Kurzansicht	E-Jahr	Zugriff	Typ	Operations
Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial 2024 . Karaa, Amel \| Bertini, Enrico \| Carelli, Valerio \| Cohen, Bruce \| Ennes, Gregory M. \| Falk, Marni J. \| Goldstein, Amy \| Gorman, Gráinne \| Haas, Richard \| Hirano, Michio \| Klopstock, Thomas \| Koenig, Mary Kay \| Kornblum, Cornelia \| Lamperti, Costanza \| Lehman, Anna \| Longo, Nicola \| Molnar, Maria Judit \| Parikh, Sumit \| Phan, Han \| Pitceathly, Robert D. S. \| Saneto, Russekk \| Scaglia, Fernando \| Servidei, Serenella \| Tarnopolsky, Mark \| Toscano, Antonio \| Van Hove, Johan L. K. \| Vissing, John \| Vockley, Jerry \| Finman, Jeffrey S. \| Abbruscato, Anthony \| Brown, David A. \| Sullivan, Alana \| Shiffer, James A. \| Mancuso, Michelango \| on behalf of the MMPOWER-3 Trial Investigators	2024		http://purl.org/ontology/bibo/Article
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