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NCT02716246
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +12 more
- Locations
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- Nationwide Children's Hospital Columbus, Ohio, United States
- Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
- Women's and Children's Hospital North Adelaide, South Australia, Australia
- Vall d'Hebron Barcelona Hospital Campus Barcelona, Spain
Recruiting
Study of Cannabidiol in Sanfilippo Syndrome
NCT06333041
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center Torrance, California, United States
NCT06614894
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +11 more
- Locations
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- Lysosomal & Rare Disorders Research & Treatment Center, Inc. Fairfax, Virginia, United States
Enrolling by Invitation
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
NCT04360265
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +13 more
- Locations
-
- Nationwide Children's Hospital Columbus, Ohio, United States
- Women's and Children's Hospital North Adelaide, South Australia, Australia
- Vall d'Hebron Barcelona Campus Barcelona, Spain
- Hospital Clínico Universitario de Santiago Santiago de Compostela, Spain
NCT02060526
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +18 more
- Locations
-
- Los Angeles Biomedical Research Torrance, California, United States
- University of Minnesota Department of Pediatrics Minneapolis, Minnesota, United States
- University of North Carolina Chapel Hill, North Carolina, United States
- Hospital Universitario Austral A Unidad de Investigacion Buenos Aires, Argentina
NCT00383448
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
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- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States