Document is current

Article History

Received: 18 December 2023

Accepted: 13 October 2024

First Online: 5 December 2024

Declarations

:

: The study was conducted in compliance with Good Epidemiology Practice guidelines, the ethical principles arising from the Declaration of Helsinki revised in 2013, and all current local regulations. Ethics Committee/Institutional Review Board approval was obtained as required by applicable site policies, national privacy regulations and other state and local laws relating to medical information. Informed consent of the parent or legal guardian was obtained as required by local law.

: Not applicable.

: BH: Is an Investigator in the RETRIEVE study and in P1-GM101 Lysogene clinical trial and in other clinical studies supported by Orphazyme/KemPharm, Azafaros, Sanofi Genzyme, Chiesi, JR Pharmaceuticals, Mallinckrodt. SB: Nothing to disclose. EM: Has received research grants and consultation fees and speakers’ honoraria from Sanofi Genzyme, Amicus, Takeda, Orphazyme, Prevail, Idorsia, Cyclo Therapeutics, Sio and Taysha. RG: Has received research grants, investigator fee, speaker honorarium, support for travel to scientific meetings, or consultation fee from Abeona, Allievex, Amicus, Avrobio, Azafaros, BioMarin, Chiesi, Cyclo, Denali, Idorsia, Inventiva, Janssen, JCR, Novartis, Paradigm, PassageBio, Pfizer, PTC, RegenxBio, Sanofi, Sigilon, Sobi, Takeda, and Ultragenyx. MP: Is an investigator in trials supported by Amicus, Glycomine, Idorsia, Maggie’s Pearl, Orphazyme/KemPharm and Shire/Takeda; receives honoraria as Editor-in-Chief of the Journal of Child Neurology (Sage), Editor of JIMD and JIMD Reports (SSIEM), and royalties as a contributor to, and section editor for, UpToDate (Wolters-Kluwer); owns stock in IntraBio. MG: Has received consulting fees from Sanofi Genzyme, and is an investigator for industrial trials from Horizon, Idorsia, Kaleido, Mallinckrodt, and Orphazyme A/S. PC: Employee of Idorsia Pharmaceuticals Ltd. LT: Employee of Idorsia Pharmaceuticals Ltd. at the time the work was performed. BS: Employee of Idorsia Pharmaceuticals Ltd. MR: Has received consulting fees from Sanofi Genzyme, and travel expenses and congress fees from Amicus.