1Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, 1 Gustave L. Levy Place, New York, NY 10029 USA
2Medical Genetics Service HCPA, Department of Genetics UFRGS, DASA and Casa Dos Raros, Porto Alegre, Brazil
3Reference Centre of Inherited Metabolic Disease in Femme Mère Enfant Hospital, Hospices Civils of Lyon, Lyon, France
4Manchester University National Health Service Trust, St Mary’s Hospital, Manchester, UK
5Institute of Clinical Science for Lysosomal Storage Disorders, SphinCS GmbH, Mainz, Germany
6University Hospital of Udine, Udine, Italy
7University Hospitals Leuven, Louvain, Belgium
8Sanofi, Bridgewater, NJ USA
9Sanofi, Cambridge, MA USA
10Sanofi, Paris, France
© The Author(s) 2023
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