237
Participants
Start Date
July 31, 2005
Primary Completion Date
May 1, 2020
Study Completion Date
May 1, 2020
Royal Children's Hospital, Brisbane, Brisbane
Women and Children's Hospital, Adelaide
Princess Margaret Hospital for Children, Subiaco
Institute of Pathology and Genetics, Metabolic Unit, Charleroi
New York University, New York
Charité-Universitätsmedizin Berlin, Berlin
Children's Hospital of Pittsburgh, Pittsburgh
Children's Hospital of Philadelphia, Philadelphia
"Università Milano Bicocca, Resp. Centro Fondazione Mariani", Monza
Universitätsklinikum Hamburg Eppendorf, Hamburg
Johns Hopkins Univeristy School of Medicine, Baltimore
Children's Hospital of the King's Daughters, Norfolk
Carolinas Medical Center, Charlotte
Fullerton Genetic Center, Asheville
Emory University, Decatur
Oberarzt Pädiatrie, Hanover
Department of Woman's and Child's Health, Padua
Indiana University School of Medicine, Indianapolis
Universitätsmedizin, Mainz
Children's Health Care, Minneapolis
University of Minnesota - Fairview University Medical Center, Minneapolis
Sioux Valey Children's Speciality Clinics, Sioux Falls
Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago
Children's Mercy Hospital, Kansas City
Hôpital Femme Mère Enfant, Bron
Tulane University Medical Center, New Orleans
Baton Rouge Clinic, Baton Rouge
Universitatsklinikum Freiburg, Klinik II, Kammin
University of Utah Medical Center, Salt Lake City
Children's Hospital of Los Angeles, Los Angeles
University of California, Irvine, Irvine
Children's Hospital and Research Center Oakland, Oakland
Azienda Ospedaliero Universitaria, Cibali
Oregon Health and Science University, Portland
Seattle Children's Hospital, Seattle
St. Joseph's Healthcare, Paterson
Westmead Hospital, Wentworthville
Royal Children's Hospital, Parkville
Hopital Necker - Enfants Malades, Paris
MidWestern Regional Hospital, Limerick
Astrid Lindgrens Children's Hospital, Stockholm
LKH-Universitätsklinik Graz, Kinderklinik, Graz
Vilnius University Hospital, Santariskiu Klinikos, Vilnius
Rotterdam University Hospital - Sophia's Children's Hospital, Rotterdam
Hospital de Sao Joao, Unidae de Doencas Metabolicas, Porto
Queen Elizabeth Hospital, University Hospitals Birmingham NHS, Birmingham
Birmingham Children's Hospital, Steelhouse, Birmingham
Great Ormond Street Hospital For Children, NHS Foundation Trust, London
St Mary's Hospital, Willink Biochemical Genetics Unit, Manchester
Salford Royal Hospital NHS Trust, Salford
Lead Sponsor
BioMarin Pharmaceutical
INDUSTRY