2026-04-06T19:11:54Z https://pub.unibl.org/oai

oai:pub.unibl.org:66635 2026-03-17T04:43:22Z

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 /unibl/sci/idNaucniRad:37266 Academic Article text 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 1027 1037 THE LANCET NEUROLOGY 1474-4422 /unibl/author/Benedikt Schoser /unibl/author/Mark Roberts /unibl/author/Barry Byrne /unibl/author/Sheela Sitaraman /unibl/author/Hai Jiang /unibl/author/Pascal Laforet /unibl/author/Antonio Toscano /unibl/author/Jeff Castelli /unibl/author/Jordi Diaz- Manera /unibl/author/Mitchell Goldman /unibl/author/Ans T Van der Ploeg /unibl/author/Drago Batkovic /unibl/author/Srilakshimi Kuchipudi /unibl/author/Tahseen Mozaffar /unibl/author/Priya S Kishnani /unibl/author/Agnes Sebok /unibl/author/Alan Pestronk https://www.unibl.org/sr-lat/fis/zaposlen/3327-aleksandra-dominovic-kovacevic /unibl/author/Aneal Khan /unibl/author/Blaž Koritnik /unibl/author/Celine Tard /unibl/author/Christopher Lindberg /unibl/author/Collin Quinn /unibl/author/Crystal Eldridge /unibl/author/Cynthia Bodkin /unibl/author/David Reyes-Leiva /unibl/author/Derralynn Hughes /unibl/author/Ela Stefanescu /unibl/author/Emmanuelle Salort-Campana /unibl/author/Ernest Butler /unibl/author/Francoise Bouhour /unibl/author/Gee Kim /unibl/author/George Konstantinos-Papadimas /unibl/author/Giancarlo Parenti /unibl/author/Halina Bartosik-Psujek /unibl/author/Hani Kushlaf /unibl/author/Hashiguchi Akihiro /unibl/author/Heather Lau /unibl/author/Helio Pedro /unibl/author/Henning Andersen /unibl/author/Hernan Amartino /unibl/author/Hideaki Shiraishi /unibl/author/Hiroshi Kobayashi /unibl/author/Ivaylo Tarnev /unibl/author/Jaime Vengoechea /unibl/author/Jennifer Avelar /unibl/author/Jin-Hong Shin /unibl/author/Jonathan Cauci /unibl/author/Jorge Alonso-Perez /unibl/author/Jozsef Janszky /unibl/author/Julie Berthy /unibl/author/Cornelia Kornblum /unibl/author/Kristina Gutschmidt /unibl/author/Kristl Claeys /unibl/author/Maria Judit-Molnar /unibl/author/Marie Wencel /unibl/author/Mark Tarnopolsky /unibl/author/Mazen Dimachkie /unibl/author/Michel Tchan /unibl/author/Miriam Freimer /unibl/author/Nikola Longo /unibl/author/Nuria Vidal-Fernandez /unibl/author/Olimpia Musumeci /unibl/author/Ozlem Goker-Alpan /unibl/author/Patrick Deegan /unibl/author/Paula Clemens /unibl/author/Richard Roxburgh /unibl/author/Robert Henderson /unibl/author/Robert Hopkin /unibl/author/Sabrina Sacconi /unibl/author/Simona Fecarotta /unibl/author/Shahram Attarian /unibl/author/Stephan Wenninger /unibl/author/Stephanie Dearmey /unibl/author/Tarekegn Hiwot /unibl/author/Thomas Burrow /unibl/author/Tobias Ruck /unibl/author/Tomo Sawada /unibl/author/Vescei Laszlo /unibl/author/Wolfgang Loscher /unibl/author/Yin-Hsiu Chien THE LANCET NEUROLOGY 1474-4422 1037 1027 /unibl/author/Benedikt Schoser /unibl/author/Mark Roberts /unibl/author/Barry Byrne /unibl/author/Sheela Sitaraman /unibl/author/Hai Jiang /unibl/author/Pascal Laforet /unibl/author/Antonio Toscano /unibl/author/Jeff Castelli /unibl/author/Jordi Diaz- Manera /unibl/author/Mitchell Goldman /unibl/author/Ans T Van der Ploeg /unibl/author/Drago Batkovic /unibl/author/Srilakshimi Kuchipudi /unibl/author/Tahseen Mozaffar /unibl/author/Priya S Kishnani /unibl/author/Agnes Sebok /unibl/author/Alan Pestronk https://www.unibl.org/sr-lat/fis/zaposlen/3327-aleksandra-dominovic-kovacevic /unibl/author/Aneal Khan /unibl/author/Blaž Koritnik /unibl/author/Celine Tard /unibl/author/Christopher Lindberg /unibl/author/Collin Quinn /unibl/author/Crystal Eldridge /unibl/author/Cynthia Bodkin /unibl/author/David Reyes-Leiva /unibl/author/Derralynn Hughes /unibl/author/Ela Stefanescu /unibl/author/Emmanuelle Salort-Campana /unibl/author/Ernest Butler /unibl/author/Francoise Bouhour /unibl/author/Gee Kim /unibl/author/George Konstantinos-Papadimas /unibl/author/Giancarlo Parenti /unibl/author/Halina Bartosik-Psujek /unibl/author/Hani Kushlaf /unibl/author/Hashiguchi Akihiro /unibl/author/Heather Lau /unibl/author/Helio Pedro /unibl/author/Henning Andersen /unibl/author/Hernan Amartino /unibl/author/Hideaki Shiraishi /unibl/author/Hiroshi Kobayashi /unibl/author/Ivaylo Tarnev /unibl/author/Jaime Vengoechea /unibl/author/Jennifer Avelar /unibl/author/Jin-Hong Shin /unibl/author/Jonathan Cauci /unibl/author/Jorge Alonso-Perez /unibl/author/Jozsef Janszky /unibl/author/Julie Berthy /unibl/author/Cornelia Kornblum /unibl/author/Kristina Gutschmidt /unibl/author/Kristl Claeys /unibl/author/Maria Judit-Molnar /unibl/author/Marie Wencel /unibl/author/Mark Tarnopolsky /unibl/author/Mazen Dimachkie /unibl/author/Michel Tchan /unibl/author/Miriam Freimer /unibl/author/Nikola Longo /unibl/author/Nuria Vidal-Fernandez /unibl/author/Olimpia Musumeci /unibl/author/Ozlem Goker-Alpan /unibl/author/Patrick Deegan /unibl/author/Paula Clemens /unibl/author/Richard Roxburgh /unibl/author/Robert Henderson /unibl/author/Robert Hopkin /unibl/author/Sabrina Sacconi /unibl/author/Simona Fecarotta /unibl/author/Shahram Attarian /unibl/author/Stephan Wenninger /unibl/author/Stephanie Dearmey /unibl/author/Tarekegn Hiwot /unibl/author/Thomas Burrow /unibl/author/Tobias Ruck /unibl/author/Tomo Sawada /unibl/author/Vescei Laszlo /unibl/author/Wolfgang Loscher /unibl/author/Yin-Hsiu Chien https://pub.unibl.org/s/cir/item/66635