| Authors: |
Mistry PK; Department of Internal Medicine, Yale University School of Medicine, New Haven, Connecticut, USA., Cassiman D; Department of Gastroenterology-Hepatology, University Hospitals Leuven, Leuven, Belgium., Jones SA; Willink Biochemical Genetics Unit, St. Mary's Hospital, Manchester University Foundation Trust, University of Manchester, Manchester, UK., Lachmann R; Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, UK., Lukina E; Department of Orphan Diseases, National Medical Research Center for Hematology, Moscow, Russian Federation., Prada CE; Division of Genetics, Genomics, and Metabolism, Ann & Robert H. Lurie Children's Hospital of Chicago, Feinberg School of Medicine, Northwestern University, Chicago, Illinois, USA., Wasserstein MP; Division of Pediatric Genetic Medicine, Children's Hospital at Montefiore and Albert Einstein College of Medicine, Bronx, New York, USA., Thurberg BL; President and Founder, Beth Thurberg Orphan Science Consulting, LLC, Newton, Massachusetts, USA., Foster MC; Global Medical Affairs: Rare Diseases, Sanofi, Cambridge, Massachusetts, USA., Patel RM; Global Scientific Communications and Publications: Rare Diseases, Sanofi, Cambridge, Massachusetts, USA., Underhill LH; Global Scientific Communications and Publications: Rare Diseases, Sanofi, Cambridge, Massachusetts, USA., Peterschmitt MJ; Research and Development, Sanofi, Cambridge, Massachusetts, USA. |