2026-04-25T11:26:39Z
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
Priya S Kishnani
creator
Barry Byrne
creator
Kristl G Claeys
creator
Jordi Diaz- Manera
creator
Mazen M Dimachkie
creator
Hani Kushlaf
creator
Tahseen Mozaffar
creator
Mark Roberts
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Benedikt Schoser
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Noemi Hummel
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Agnieszka Kopiec
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Fred Holdbrook
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Simon Shohet
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Antonio Toscano
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Agnes Sebok
creator
Alan Pestronk
creator
Александра Доминовић-Ковачевић
creator
Aneal Khan
creator
Blaž Koritnik
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Celine Tard
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Christopher Lindberg
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Collin Quinn
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Crystal Eldridge
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Cynthia Bodkin
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David Reyes-Leiva
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Derralynn Hughes
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Ela Stefanescu
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Emmanuelle Salort-Campana
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Ernest Butler
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Francoise Bouhour
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Gee Kim
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George Konstantinos-Papadimas
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Giancarlo Parenti
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Halina Bartosik-Psujek
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Hashiguchi Akihiro
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Heather Lau
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Helio Pedro
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Henning Andersen
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Hernan Amartino
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Hideaki Shiraishi
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Hiroshi Kobayashi
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Ivaylo Tarnev
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Jaime Vengoechea
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Jennifer Avelar
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Jin-Hong Shin
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Jonathan Cauci
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Jorge Alonso-Perez
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Jozsef Janszky
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Julie Berthy
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Cornelia Kornblum
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Kristina Gutschmidt
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Maria Judit-Molnar
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Marie Wencel
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Mark Tarnopolsky
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Michel Tchan
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Miriam Freimer
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Nikola Longo
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Nuria Vidal-Fernandez
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Olimpia Musumeci
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Ozlem Goker-Alpan
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Patrick Deegan
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Paula R Clemens
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Richard Roxburgh
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Robert Henderson
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Robert Hopkin
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Sabrina Sacconi
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Simona Fecarotta
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Shahram Attarian
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Stephan Wenninger
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Stephanie Dearmey
creator
Tarekegn Hiwot
creator
Thomas Burrow
creator
Tobias Ruck
creator
Tomo Sawada
creator
Vescei Laszlo
creator
Wolfgang Loscher
creator
Yin-Hsiu Chien
creator
Academic Article
text
/unibl/sci/idNaucniRad:37283
info:doi/10.1186/s41687-024-00805-w
Journal of patient - reported outcomes
2509-8020
https://www.pub.unibl.org/s/cir/item/66642
2024-11
66642