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Eugen Mengel is a Principal Investigator on the venglustat LEAP trial and reports receiving fees and consultant honoraria from Actelion, Alexion, Avrobio, Cyclo Therapeutics, Freeline Therapeutics, Idorsia, Orphazyme A\/S, Sanofi, and Takeda. Mary Wallace was the Clinical Study Coordinator on the venglustat LEAP trial. Ruth Pulikottil-Jacob, Camille Rochmann, Isabela Batsu, Riliang Zheng, and Alaa Hamed are employees of Sanofi; Isabela Batsu and Riliang Zheng hold shares and\/or stock options in this company. James Turnbull and Robert Krupnick are employees of IQVIA, Inc., which received fees for this study. Chad Gwaltney is an employee of Gwaltney Consulting and was a paid consultant of Sanofi for this study; however, this author did not receive any payment related to authoring this manuscript.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of Interest"}},{"value":"This standalone qualitative study was not included in the LEAP trial protocol. The study was approved by a third-party institutional review board (Advarra, Columbia, MD) and was conducted in accordance with the Helsinki Declaration of 1964 and its later amendments. Patients and caregivers provided informed consent to participate online or on paper after being approached by the research coordinator from a participating clinical trial site in the US. Patients and caregivers were also asked for verbal confirmation of their consent before the start of their interviews.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval"}}]}}