12,000
Participants
Start Date
December 8, 2016
Primary Completion Date
December 8, 2030
Study Completion Date
December 8, 2030
RECRUITING
University of Pittsburgh Medical Center, Pittsburgh
RECRUITING
The Children's Hospital of Philadelphia, Philadelphia
RECRUITING
University of Pennsylvania, Philadelphia
RECRUITING
Children's National Medical Center, Washington D.C.
RECRUITING
Kennedy Krieger Institute, Baltimore
RECRUITING
Atrium Health Wake Forest Baptist, Winston-Salem
RECRUITING
Emory University (Children's Healthcare of Atlanta), Atlanta
RECRUITING
Nationwide Children's Hospital, Columbus
RECRUITING
Akron Children's Hospital, Akron
RECRUITING
University of Minnesota, Minneapolis
RECRUITING
Mayo Clinic, Rochester
RECRUITING
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago
RECRUITING
Baylor College of Medicine (Texas Children's Hospital), Houston
RECRUITING
University of Utah (Primary Children's Hospital), Salt Lake City
RECRUITING
Children's Hospital of Los Angeles, Los Angeles
RECRUITING
University of California, San Diego (Rady Children's Hospital), San Diego
RECRUITING
Children's Hospital of Orange County, Orange
RECRUITING
UCSF Benioff Children's Hospital, San Francisco
RECRUITING
Stanford University (Lucile Packard Children's Hospital), Palo Alto
RECRUITING
University of California, Davis (UC Davis Health), Sacramento
RECRUITING
Seattle Children's Hospital, Seattle
RECRUITING
Massachusetts General Hospital (MGH), Boston
RECRUITING
UT Health Houston, Houston
National Institutes of Health (NIH)
NIH
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
National Center for Advancing Translational Sciences (NCATS)
NIH
Biogen
INDUSTRY
Eli Lilly and Company
INDUSTRY
Myrtelle Inc.
INDUSTRY
Orchard Therapeutics Ltd.
UNKNOWN
Passage Bio, Inc.
INDUSTRY
Synaptix Biotherapeutics Ltd.
UNKNOWN
Takeda
INDUSTRY
Boehringer Ingelheim
INDUSTRY
Ionis Pharmaceuticals, Inc.
INDUSTRY
Sanofi Winthrop Industrie
UNKNOWN
Sana Biotechnology
INDUSTRY
Yaya Foundation for 4H Leukodystrophy
UNKNOWN
University of Pennsylvania
OTHER
United MSD Foundation
UNKNOWN
Foundation to Fight H-ABC
UNKNOWN
Calliope Joy Foundation
UNKNOWN
Don't Forget Me Foundation
UNKNOWN
Children's Hospital of Philadelphia
OTHER