Received: 21 July 2021
Accepted: 6 December 2021
First Online: 20 December 2021
: Use of the de-identified data examined in this initiative to study patient characteristics and outcomes in retrospective, non-interventional, secondary analyses has been determined to be exempt from institutional review board (IRB) oversight by an independent IRB.
: Not applicable.
: JJ reports honoria from Sanofi Genzyme, Chiesi Pharma, Amicus, Abbott, CHFSolutions, Audentes, AstraZeneca, and IPS Heart. AS reports employment and stock options at OM1, Inc. HL reports grant/research support and advisory board membership with grant, honoraria, and reimbursed travel/food for Amicus and Biomarin, advisory board membership with honoria, reimbursed travel/food for Chieisi and Actellion, grant/research support, consultant, advisory board membership, fellowship grant with research support, honoraria, and reimbursed travel/food from Sanofi, grant/research support, advisory board membership with honoria, reimbursed travel/food from Takeda, grant/research support, consultant, advisory board membership, employment with honoria, reimbursed travel/food, and salary at Ultragenyx, grant/research support from Mallinckrodt, Intrabio, Pfizer, Protalix, Ovid Therapeutics, and Denali, grant research support, consultant at Prevail Therapeutics and Aspa Therapeutics. Advisory board membership with honoria for Taysha, Advisory board membership and lecturer with honoria at National Gaucher Foundation and National Fabry Disease Foundation, advisory board member, lecturer, and grant support from National Tay Sachs and Allied Diseases, lecturer with honoraria for FSIG, advisory board member, researcher for Adult Polyglucosan Body Disorders Research Foundation. MN reports salary, stock, and benefits from Amicus Therapeutics, Inc. JG reports salary, stock, and stock options at Amicus Therapeutics, Inc. JZ reports employment and stock options at OM1, Inc. CB reports employment and stock options at OM1, Inc. GC reports stock options from OM1, Inc., employment at Brigham and Women’s Hospital, consultant activities at and stock options from Allena Pharmaceuticals, holding a grant from Decibel Therapeutics and GSK, and consultant activities for Dicerna, Shire-Takeda, and AstraZeneca. RG reports employment and stock options at OM1, Inc. DW reports stock ownership in Reata Pharma, advisory-board membership at Chiesi Pharma, and consultant activities for Amicus Biotherapeutics and Protalix Biotherapuetics.