Unosi

• 104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-labek extension study (ATB200-07) (2024)

  B. Schoser, P. Kishnani, D. Batkovic, B. Byrne, K. Claeys, J. Diaz- Manera, P. Laforet, M. Roberts, A. Toscano, A. Van der Ploeg, J. Castelli, M. Goldman, F. Holdbrook, S. Sitaraman, Y. Wasfi, T. Mozaffar, A. Sebok, A. Pestronk, A. Dominović-Kovačević, A. Khan, B. Koritnik, C. Tard, C. Lindberg, C. Quinn, C. Eldridge, C. Bodkin, D. Reyes-Leiva, D. Hughes, E. Stefanescu, E. Salort-Campana, E. Butler, F. Bouhour, G. Kim, G. Konstantinos-Papadimas, G. Parenti, H. Bartosik-Psujek, H. Kushlaf, H. Akihiro, H. Lau, H. Pedro, H. Andersen, H. Amartino, H. Shiraishi, H. Kobayashi, I. Tarnev, J. Vengoechea, J. Avelar, J. Shin, J. Nevin, J. Cauci, J. Alonso-Perez, J. Janszky, J. Berthy, C. Kornblum, K. Gutschmidt, M. Judit-Molnar, M. Wencel, M. Tarnopolsky, M. Boentert, M. Tchan, M. Freimer, N. Longo, N. Abreu, N. Vidal-Fernandez, O. Musumeci, O. Goker-Alpan, P. Deegan, P. Clemens, R. Roxburgh, R. Henderson, R. Hopkin, S. Sacconi, S. Fecarotta, S. Attarian, S. Wenninger, S. Dearmey, T. Hiwot, T. Burrow, T. Ruck, T. Sawada, V. Laszlo, W. Loscher, Y. Chien, 104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-labek extension study (ATB200-07), JOURNAL OF NEUROLOGY, Vol. 271, pp. 2810 - 2823, 2024