Lead Sponsor: National Institute of Neurological Disorders and Stroke (NINDS)

Lead Sponsor: National Institute of Neurological Disorders and Stroke (NINDS)

Lead Sponsor: University of Iowa

Collaborators: National Institute of Neurological Disorders and Stroke (NINDS), Children's Hospital of Philadelphia, University of Pennsylvania, Cedars-Sinai Medical Center, University of Rochester, University of Minnesota, Stanford University, University of Miami, Massachusetts General Hospital, University of Michigan, University of Colorado, Denver, Johns Hopkins University, Children's National Research Institute, The Hospital for Sick Children, Connecticut Children's Medical Center, Seattle Children's Hospital, Sydney Children's Hospitals Network, Muscular Dystrophy Association, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Nemours Children's Clinic, St. Jude Children's Research Hospital, King's College Hospital NHS Trust