1. Department of Pediatrics and Clinical and Translational Science Institute, Children’s National Medical Center; and School of Medicine and Health Sciences, George Washington University, Washington, DC 20010, USA.
2. Center for Clinical and Translational Research, The Research Institute at Nationwide Children’s Hospital, and the Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA.
3. Department of Pediatrics and Tufts Clinical and Translational Science Institute, The Floating Hospital for Children at Tufts Medical Center, Boston, MA 02111, USA.
4. The Eunice Kennedy Shriver NICHD, Bethesda, MD 20892, USA.
5. Office of Orphan Products Development, FDA, Silver Spring, MD 20993, USA.
6. CTSA, NCATS, Bethesda, MD 20892, USA.