Schoser, Benedikt http://orcid.org/0000-0002-2757-8131
Kishnani, Priya S.
Bratkovic, Drago
Byrne, Barry J.
Claeys, Kristl G.
Díaz-Manera, Jordi
Laforêt, Pascal
Roberts, Mark
Toscano, Antonio
van der Ploeg, Ans T.
Castelli, Jeff
Goldman, Mitchell
Holdbrook, Fred
Sitaraman Das, Sheela
Wasfi, Yasmine
Mozaffar, Tahseen
,
Sebok, Agnes
Pestronk, Alan
Dominovic-Kovacevic, Aleksandra
Khan, Aneal
Koritnik, Blaž
Tard, Celine
Lindberg, Christopher
Quinn, Colin
Eldridge, Crystal
Bodkin, Cynthia
Reyes-Leiva, David
Hughes, Derralynn
Stefanescu, Ela
Salort-Campana, Emmanuelle
Butler, Ernest
Bouhour, Francoise
Kim, Gee
Papadimas, George Konstantinos
Parenti, Giancarlo
Bartosik-Psujek, Halina
Kushlaf, Hani
Akihiro, Hashiguchi
Lau, Heather
Pedro, Helio
Andersen, Henning
Amartino, Hernan
Shiraishi, Hideaki
Kobayashi, Hiroshi
Tarnev, Ivaylo
Vengoechea, Jaime
Avelar, Jennifer
Shin, Jin-Hong
Nevin, John
Cauci, Jonathan
Alonso-Pérez, Jorge
Janszky, Jozsef
Berthy, Julie
Kornblum, Cornelia
Gutschmidt, Kristina
Molnar, Maria Judit
Wencel, Marie
Tarnopolsky, Mark
Boentert, Matthias
Tchan, Michel
Freimer, Miriam
Longo, Nicola
Abreu, Nicolas
Vidal-Fernandez, Nuria
Musumeci, Olimpia
Goker-Alpan, Ozlem
Deegan, Patrick
Clemens, Paula R.
Roxburgh, Richard
Henderson, Robert
Hopkin, Robert
Sacconi, Sabrina
Fecarotta, Simona
Attarian, Shahram
Wenninger, Stephan
Dearmey, Stephanie
Hiwot, Tarekegn
Burrow, Thomas
Ruck, Tobias
Sawada, Tomo
Laszlo, Vescei
Löscher, Wolfgang
Chien, Yin-Hsiu
Clinical trials referenced in this document:
Documents that mention this clinical trial
104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)
https://doi.org/10.1007/s00415-024-12236-0
Funding for this research was provided by:
Amicus Therapeutics
Universitätsklinik München
Article History
Received: 23 October 2023
Revised: 31 January 2024
Accepted: 1 February 2024
First Online: 28 February 2024
Declarations
:
: Benedikt Schoser has received unrestricted research grants from Amicus Therapeutics Inc., Astellas, Roche, Marigold Foundation, AMDA Foundation and speaker’s honoraria from Amicus Therapeutics Inc., Alexion, Kedrion, Sanofi. He has participated as a scientific advisor for Amicus Therapeutics Inc., Argenx, Astellas, Bayer, Maze, Pepgen, Sanofi, Spark, and Taysha. He declares no stocks or shares. Drago Bratkovic declares no competing interests. Barry J. Byrne has participated as a consultant/advisory board member for Pfizer, Amicus Therapeutics, Inc., and Sanofi. He also owns stocks in Lacerta Therapeutics. Kristl G. Claeys received research funding from Alnylam, Biogen, Pfizer, Roche, Sanofi-Genzyme; received advisory board member honoraria from Alexion, Alnylam, Amicus, ArgenX, Biogen, Ipsen, Janssen Pharmaceutics, Lupin, Pfizer, Roche, Sanofi-Genzyme and UCB and is Chairholder of the Emil von Behring Chair for Neuromuscular and Neurodegenerative Disorders by CSL Behring. Jordi Diaz-Manera has received consulting fees/honoraria from Sarepta, Sanofi and Audentes and grant support from Sanofi, Spark and Boehringer Ingelheim. He has received payment for speaking from Sanofi, Sarepta and Lupin. Priya S. Kishnani has received research/grant support from Sanofi Genzyme and Amicus Therapeutics Inc. and has received consulting fees and honoraria from Sanofi Genzyme, Amicus Therapeutics, Inc., Maze Therapeutics, Bayer and Asklepios Biopharmaceutical, Inc. (AskBio). She is a member of the Pompe and Gaucher Disease Registry Advisory Board for Sanofi Genzyme, Pompe Disease Advisory Board for Amicus Therapeutics Inc., and Advisory Board for Baebies. Priya S. Kishnani has equity with Maze Therapeutics and has held equity in Asklepios Biopharmaceuticals and may receive milestone payments related to that equity in the future. Pascal Laforet is a consultant/advisory board member for Amicus Therapeutics Inc., Sanofi Genzyme and Spark Therapeutics. He has received travel expenses from Sanofi Genzyme and Spark Therapeutics. Mark Roberts has received honorarium for educational symposia from Sanofi Genzyme and Amicus and for participation on advisory boards for Sanofi, and Amicus. Antonio Toscano received honorarium for educational talks from Sanofi Genzyme and Amicus and for participation on advisory boards for Sanofi, Amicus, Aro and Spark. He is a member of the European Reference Network for Neuromuscular Disorders (EU-NMD), Project ID 739543. Ans T. van der Ploeg is an advisory board member of Amicus Therapeutics Inc., BioMarin, Sanofi Genzyme, and Spark Therapeutics. She has provided consultancies for Amicus Therapeutics Inc., BioMarin, Sanofi Genzyme, and Spark Therapeutics and has contracted research for Amicus Therapeutics Inc., BioMarin, Sanofi Genzyme, and Spark Therapeutics. All collaborations are done under an agreement between Erasmus MC and these industries. Tahseen Mozaffar has participated in an advisory capacity for Abbvie, Alexion, Amicus Therapeutics, Inc., Annji, Argenx, Arvinas, Audentes, Cabaletta, Maze Therapeutics, Momenta, Ra Pharmaceuticals, Sanofi Genzyme, Sarepta, Spark Therapeutics, and UCB, and has participated in the speaker’s bureau for Sanofi Genzyme. He is a member of the medical advisory board for the Myositis Association, Neuromuscular Disease Foundation, Myasthenia Gravis Foundation of California and Myasthenia Gravis Foundation of America. He has received research funding from the Myositis Association, the Muscular Dystrophy Association, the NIH and from the following sponsors: Alexion, Amicus Therapeutics, Inc., Annji, Argenx, Audentes, Bristol-Myers Squib, Cabaletta, Cartesian Therapeutics, Grifols, Momenta, Ra Pharmaceuticals, Sanofi Genzyme, Spark Therapeutics, UCB, and Valerion. He is a member of the Data safety monitoring board for Acceleron, Applied Therapeutics, Sarepta, and the NIH. Jeff Castelli, Mitchell Goldman, Fred Holdbrook, Sheela Sitaraman Das, and Yasmine Wasfi are employees of, and hold stocks and shares in, Amicus Therapeutics, Inc.
: This study was conducted in accordance with ethical standards as laid down in the 1964 Declaration of Helsinki and its later amendments, and in compliance with the United States (US) Food and Drug Administration (FDA) regulations in 21 Code of Federal Regulations 56, European Union (EU) Clinical Trials Regulation 536/2014, and the International Council for Harmonisation Good Clinical Practice (GCP) guidelines. For each study site, the clinical study protocol (and any amendments) and informed consent form were reviewed and approved by the appropriate Independent Ethics Committee (IEC)/Institutional Review Board (IRB).
: All patients provided signed informed consent before any study-related procedures were performed. In Japan, the patient’s parental guardian (or legal representative) also had to sign the informed consent form for patients under 20 years of age.