Florida

• University of Miami*
  
  *This site does not see or enroll patients

Iowa

• University of Iowa, Iowa City

Maryland

• Johns Hopkins University, Baltimore

Michigan

• Wayne State University, Detroit

New York

• University of Rochester

Pennsylvania

• University of Pennsylvania School of Medicine, Philadelphia
  
  
• Children's Hospital of Philadelphia (CHOP)

Tennessee

• Vanderbilt University, Nashville

Washington

• University of Washington, Seattle

Australia

• The Children's Hospital at Westmead, Sydney, NSW, Australia

Italy

• C. Besta Neurological Institute, Milan

United Kingdom

• Center for Neuromuscular Diseases, National Hospital for Neurology and Neurosurgery, London
  
  
• Great Ormond St Hospital for Children, London