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National Institute for Health and Welfare, Public Health Genomics Unit, Helsinki, Finland Institute for Molecular Medicine Finland, FIMM, Biomedicum Helsinki, Helsinki, Finland Pediatric Storage Disorders Laboratory, Department of Neuroscience and Centre for the Cellular Basis of Behaviour, James Black Centre, King's Health Partners Centre for Neurodegeneration Research, Institute of Psychiatry, King's College London, London, UK Finnish Medicines Agency, Helsinki, Finland and University of Helsinki, Helsinki, Finland