Unosi

• Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient - reported outcome measures in patients with late-onset Pompe disease (2024-11)

  P. Kishnani, B. Byrne, K. Claeys, J. Diaz- Manera, M. Dimachkie, H. Kushlaf, T. Mozaffar, M. Roberts, B. Schoser, N. Hummel, A. Kopiec, F. Holdbrook, S. Shohet, A. Toscano, 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. Akihiro, H. Lau, H. Pedro, H. Andersen, H. Amartino, H. Shiraishi, H. Kobayashi, I. Tarnev, J. Vengoechea, J. Avelar, J. Shin, J. Cauci, J. Alonso-Perez, J. Janszky, J. Berthy, C. Kornblum, K. Gutschmidt, M. Judit-Molnar, M. Wencel, M. Tarnopolsky, M. Tchan, M. Freimer, N. Longo, 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, Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient - reported outcome measures in patients with late-onset Pompe disease, Journal of patient - reported outcomes, Vol. 13;8(1):132, Nov, 2024

• Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease(PROPEL): an international randomised,double-blind, parallel-group,phase 3 trial (2021-12)

  B. Schoser, M. Roberts, B. Byrne, S. Sitaraman, H. Jiang, P. Laforet, A. Toscano, J. Castelli, J. Diaz- Manera, M. Goldman, A. Van der Ploeg, D. Batkovic, S. Kuchipudi, T. Mozaffar, P. Kishnani, 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. Cauci, J. Alonso-Perez, J. Janszky, J. Berthy, C. Kornblum, K. Gutschmidt, K. Claeys, M. Judit-Molnar, M. Wencel, M. Tarnopolsky, M. Dimachkie, M. Tchan, M. Freimer, N. Longo, 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, Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease(PROPEL): an international randomised,double-blind, parallel-group,phase 3 trial, THE LANCET NEUROLOGY, Vol. 20(12), pp. 1027 - 1037, Dec, 2021