Author information:
(1)Department of Haematology, Royal Free London NHS Foundation Trust and 
University College London, London, UK.
(2)Department of Nephrology, Royal Melbourne Hospital, Parkville, Victoria, 
Australia.
(3)Section of Vitreoretinal Surgery & Diseases, Emory University, Atlanta, 
Georgia, USA.
(4)Division of Nephrology and Dialysis, Department of Medicine III, Medical 
University of Vienna, Vienna, Austria.
(5)Doernbecher Children's Hospital, Oregon Health & Science University, 
Portland, Oregon, USA.
(6)Infusion Associates, Grand Rapids, Missouri, USA.
(7)Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
(8)Department of Pediatrics, Osaka City University Hospital, Osaka-shi, Japan.
(9)Charles Dent Metabolic Unit, National Hospital for Neurology and 
Neurosurgery, London, UK.
(10)Jikei University Hospital, Tokyo, Japan.
(11)Departmento Cuore e vasi, A.O.U. Careggi Firenze, Firenze, Italy.
(12)Osaka City University Hospital, Osaka-shi, Japan.
(13)Lysosmal Disorders Unit, Department of Medicine, Addenbrooke's Hospital, 
Cambridge, UK.
(14)Genetics Center MS716, Children's Hospital of Wisconsin, Milwaukee, 
Wisconsin, USA.
(15)Univeritair Ziekenhaus Anterpen, Edegem, Belgium.
(16)Division of Medical Genetics, University of Versailles, Paris-Saclay 
University and Assistance Publique-Hôpitaux de Paris, Paris, France.
(17)O & O Alpan LLC, Springfield, Virginia, USA.
(18)Service de Médecine, Hôpital Claude Huriez-CHRU Lille, Lille, France.
(19)Department of Endocrinology and Metabolic Medicine, Salford Royal NHS 
Foundation Trust, Salford, UK.
(20)Hospital das Clínicas FMUSP-Ribeirão Preto, São Paulo, Ribeirão Preto, 
Brazil.
(21)Niigata University Graduate School of Medicine and Dental Sciences, Niigata, 
Japan.
(22)Royal Perth Hospital, Perth, New South Wales, Australia.
(23)Department of Genetics, Emory University School of Medicine, Atlanta, 
Georgia, USA.
(24)Clinical Research Division, Hôpital du Sacré-Coeur de Montreal, University 
of Montreal, Montreal, Quebec, Canada.
(25)Institute of Metabolic Disease, Baylor Research Institute, Dallas, Texas, 
USA.
(26)Agility Clinical Inc., Carlsbad, California, USA.
(27)Amicus Therapeutics Inc., Cranbury, New Jersey, USA.
(28)CymaBay Therapeutics, Inc., Newark, California, USA.
(29)TranscripTx, Inc., Sunnyvale, California, USA.
(30)Parkinson's Institute and Clinical Center, Sunnyvale, California, USA.
(31)Department of Medical Endocrinology, Rigshospital, Copenhagen University 
Hospital, Copenhagen, Denmark.