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

Schoser B.; Roberts M.; Byrne B.J.; Sitaraman S.; Jiang H.; Lafor?t P.; Toscano A.; Castelli J.; D?az-Manera J.; Goldman M.; van der Ploeg A.T.; Bratkovic D.; Kuchipudi S.; Mozaffar T.; Kishnani P.S.; Sebok A.; Pestronk A.; Dominovic-Kovacevic A.; Khan A.; Koritnik B.; Tard C.; Lindberg C.; Quinn C.; Eldridge C.; Bodkin C.; Reyes-Leiva D.; Hughes D.; Stefanescu E.; SALORT-CAMPANA E.; Butler E.; Bouhour F.; Kim G.; Konstantinos Papadimas G.; Parenti G.; Bartosik-Psujek H.; Kushlaf H.; Akihiro H.; Lau H.; Pedro H.; Andersen H.; Amartino H.; Shiraishi H.; Kobayashi H.; Tarnev I.; Vengoechea J.; Avelar J.; Shin J.-H.; Cauci J.; Alonso-P?rez J.; Janszky J.; Berthy J.; Cornelia K.; Gutschmidt K.; Claeys K.; Judit Molnar M.; Wencel M.; Tarnopolsky M.; Dimachkie M.; Tchan M.; Freimer M.; Longo N.; Vidal-Fernandez N.; Musumeci O.; Goker-Alpan O.; Deegan P.; Clemens P.R.; Roxburgh R.; Henderson R.; Hopkin R.; Sacconi S.; Fecarotta S.; Attarian S.; Wenninger S.; Dearmey S.; Hiwot T.; Burrow T.; Ruck T.; Sawada T.; Laszlo V.; L?scher W.; YIN-HSIU CHIEN; PROPEL Study Group

Please use this identifier to cite or link to this item: https://scholars.lib.ntu.edu.tw/handle/123456789/602263

Title:	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
Authors:	Schoser B. Roberts M. Byrne B.J. Sitaraman S. Jiang H. Lafor?t P. Toscano A. Castelli J. D?az-Manera J. Goldman M. van der Ploeg A.T. Bratkovic D. Kuchipudi S. Mozaffar T. Kishnani P.S. Sebok A. Pestronk A. Dominovic-Kovacevic A. Khan A. Koritnik B. Tard C. Lindberg C. Quinn C. Eldridge C. Bodkin C. Reyes-Leiva D. Hughes D. Stefanescu E. SALORT-CAMPANA E. Butler E. Bouhour F. Kim G. Konstantinos Papadimas G. Parenti G. Bartosik-Psujek H. Kushlaf H. Akihiro H. Lau H. Pedro H. Andersen H. Amartino H. Shiraishi H. Kobayashi H. Tarnev I. Vengoechea J. Avelar J. Shin J.-H. Cauci J. Alonso-P?rez J. Janszky J. Berthy J. Cornelia K. Gutschmidt K. Claeys K. Judit Molnar M. Wencel M. Tarnopolsky M. Dimachkie M. Tchan M. Freimer M. Longo N. Vidal-Fernandez N. Musumeci O. Goker-Alpan O. Deegan P. Clemens P.R. Roxburgh R. Henderson R. Hopkin R. Sacconi S. Fecarotta S. Attarian S. Wenninger S. Dearmey S. Hiwot T. Burrow T. Ruck T. Sawada T. Laszlo V. L?scher W. YIN-HSIU CHIEN PROPEL Study Group
Issue Date:	2021
Publisher:	Elsevier Ltd
Journal Volume:	20
Journal Issue:	12
Start page/Pages:	1027-1037
Source:	The Lancet Neurology
URI:	https://www.scopus.com/inward/record.uri?eid=2-s2.0-85119194577&doi=10.1016%2fS1474-4422%2821%2900331-8&partnerID=40&md5=5b6e810d3470dfcbb2e5d83bff96c54e https://scholars.lib.ntu.edu.tw/handle/123456789/602263
ISSN:	1474-4422
DOI:	10.1016/S1474-4422(21)00331-8
Appears in Collections:	醫學系

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