University of Pittsburgh, Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA, USA. Electronic address: gerard.vockley@chp.edu. Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA. Boston Children's Hospital, Boston, MA, USA. University of Utah, Salt Lake City, UT, USA. Vanderbilt University Medical Center, Nashville, TN, USA. University of South Florida, Morsani College of Medicine, Tampa, FL, USA. Children's National Medical Center, Washington, DC, USA. Great Ormond Street Hospital, UCL Institute of Child Health, London, UK. Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, UK. University of Montana, Missoula, MT, USA. Ultragenyx Pharmaceutical Inc., Novato, CA, USA.