2026-04-25T03:06:40Z
https://www.pub.unibl.org/oai
oai:pub.unibl.org:66642
2026-03-17T04:43:20Z
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient - reported outcome measures in patients with late-onset Pompe disease
/unibl/sci/idNaucniRad:37283
https://dx.doi.org/10.1186/s41687-024-00805-w
Academic Article
text
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
Journal of patient - reported outcomes
2509-8020
/unibl/author/Priya S Kishnani
/unibl/author/Barry Byrne
/unibl/author/Kristl G Claeys
/unibl/author/Jordi Diaz- Manera
/unibl/author/Mazen M Dimachkie
/unibl/author/Hani Kushlaf
/unibl/author/Tahseen Mozaffar
/unibl/author/Mark Roberts
/unibl/author/Benedikt Schoser
/unibl/author/Noemi Hummel
/unibl/author/Agnieszka Kopiec
/unibl/author/Fred Holdbrook
/unibl/author/Simon Shohet
/unibl/author/Antonio Toscano
/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/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/Maria Judit-Molnar
/unibl/author/Marie Wencel
/unibl/author/Mark Tarnopolsky
/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 R 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
Journal of patient - reported outcomes
2509-8020
/unibl/author/Priya S Kishnani
/unibl/author/Barry Byrne
/unibl/author/Kristl G Claeys
/unibl/author/Jordi Diaz- Manera
/unibl/author/Mazen M Dimachkie
/unibl/author/Hani Kushlaf
/unibl/author/Tahseen Mozaffar
/unibl/author/Mark Roberts
/unibl/author/Benedikt Schoser
/unibl/author/Noemi Hummel
/unibl/author/Agnieszka Kopiec
/unibl/author/Fred Holdbrook
/unibl/author/Simon Shohet
/unibl/author/Antonio Toscano
/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/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/Maria Judit-Molnar
/unibl/author/Marie Wencel
/unibl/author/Mark Tarnopolsky
/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 R 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://www.pub.unibl.org/s/cir/item/66642