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Authors
Freihuber, Cécile
Dahmani-Rabehi, Bahia
Brassier, Anaïs
Broué, Pierre
Cances, Claude
Chabrol, Brigitte
Eyer, Didier
Labarthe, François
Latour, Philippe
Levade, Thierry
Pichard, Samia
Sevin, Caroline
Vanier, Marie T.
Héron, Bénédicte http://orcid.org/0000-0002-7707-4521
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Article History
Received: 10 April 2023
Accepted: 6 July 2023
First Online: 21 July 2023
Declarations
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: This study was approved by the <i>“Comité de protection des personnes” (CPP)</i> in La Salpétrière Hospital (local ethic committee). For each living patient, an informed consent letter from the CRML (Reference Center for Lysosomal Diseases) was sent to parents by the referring clinician.
: Not applicable.
: CF received grants from Actelion Pharmaceuticals. BH received honoraria as Member of Scientific Boards from Actelion Pharmaceuticals and Orphazyme. FL reports advisory board participation for AlfaSigma and Sanofi Genzyme, and consulting fees from AlfaSigma, Biomarin and Sanofi Genzyme. MTV received honoraria as Member of Scientific Boards from Actelion Pharmaceuticals and Orphazyme, and travel from Actelion Pharmaceuticals. PL received funding from Actelion Pharmaceuticals 2008–2018 for travels and practitioner’s trainings.
