1. Department of Neurology, Division of Neuromuscular Medicine, Stanford University School of Medicine, Palo Alto, CA, USA
2. Department of Pediatrics, Duke University Medical Center, Durham, NC, USA
3. Sanofi, Chilly-Mazarin, France
4. Sanofi, Cambridge, MA, USA
5. Clinical and Metabolic Genetics, Dell Children’s Medical Group, Austin, TX, USA
6. Departments of Pediatrics and Medicine and Biochemical Genetics Program, Seattle Children’s Hospital/University of Washington, Seattle, WA, USA
7. University of Missouri Child Health, Columbia, MO, USA
8. Departments of Pathology and Pediatrics, New York Medical College, Valhalla, NY, USA
9. Division of Human Genetics, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA
10. Current address: Division of Human Genetics, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA
11. Current address: University of Cincinnati College of Medicine, Cincinnati, OH, USA
12. Division of Genetic, Genomic, and Metabolic Disorders, Central Michigan University and Children’s Hospital of Michigan, Detroit, MI, USA
13. Rare Disease Institute, Children’s National Hospital, Washington, DC, USA
14. Current address: Division of Medical Genetics, Child Health Research Center, Torrance, CA, USA