Document is current

Article History

Received: 5 April 2024

Accepted: 26 October 2024

First Online: 13 November 2024

Declarations

:

: PROPEL was approved by the appropriate independent ethics committees and institutional review boards at each study site and was conducted according to international guidelines for clinical studies, such as the Declaration of Helsinki and Good Clinical Practice Guidelines. All participants provided written informed consent before participating in the study.

: Not applicable.

: KGC received research funding from Alnylam, Biogen, Pfizer, Roche, Sanofi Genzyme; advisory board member honoraria from Alexion, Alnylam, Amicus Therapeutics, Inc., 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. BJB reports consultant/advisory board membership for Pfizer, Amicus Therapeutics, Inc., and Sanofi; and owns stocks in Lacerta Therapeutics. JD-M reports consulting fees/honoraria from Sarepta, Sanofi, Audentes; has received grant support from Sanofi, Spark and Boehringer Ingelheim; and payment for speaking from Sanofi, Sarepta and Lupin. MMD serves or recently served as a consultant for Abata/Third Rock, Abcuro, Amicus Therapeutics, Inc., argenx, Astellas, Cabaletta Bio, Catalyst, CNSA, Covance/Labcorp, CSL Behring, Dianthus, Horizon, EMD Serono/Merck, Ig Society, Inc, Janssen Pharmaceuticals, Medlink, Octapharma, Priovant, Sanofi Genzyme, Shire Takeda, TACT/Treat NMD, UCB Biopharma, Valenza Bio and Wolters Kluwer Health/UpToDate; and has received research grants or contracts, or educational grants from Alexion/AstraZeneca, Alnylam Pharmaceuticals, Amicus Therapeutics, Inc., argenx, Bristol-Myers Squibb, Catalyst, CSL Behring, FDA/OOPD, GlaxoSmithKline, Genentech, Grifols, Mitsubishi Tanabe Pharma, MDA, NIH, Novartis, Octapharma, Orphazyme, Ra Pharma/UCB, Sanofi Genzyme, Sarepta Therapeutics, Shire Takeda, Spark Therapeutics, The Myositis Association, and UCB Biopharma/RaPharma. PSK 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., JCR Pharmaceuticals, 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. She has held equity in Asklepios Biopharmaceuticals and may receive milestone payments related to that equity in the future. HK served as a consultant for Alexion AstraZeneca Rare disease, argenx, UCB, Immunovant, Sanofi, and has received research grants or educational grants from Sanofi, MDA, and Healey ALS platform trial. TM 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. 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. MR has received honoraria for educational symposia from Sanofi Genzyme and Amicus Therapeutics, Inc., and for participation on advisory boards for Sanofi, and Amicus Therapeutics, Inc. BS 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, and Sanofi. He has participated as a scientific adviser for Amicus Therapeutics, Inc., argenx, Astellas, Bayer, Maze, Pepgen, Sanofi, and Spark. He declares no stocks or shares. NH and AK are employees of Certara, which is a paid consultant to Amicus Therapeutics, Inc. FH and SS are employees of and hold stock in Amicus Therapeutics, Inc. AT has received honorarium for educational talks from Sanofi Genzyme and Amicus Therapeutics, Inc. and for participation on advisory boards for Sanofi, Amicus Therapeutics, Inc., Aro and Spark. He is a member of the European Reference Network for Neuromuscular Disorders (EU-NMD)– Project ID 739543.