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NCT02716246
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +12 more
- Locations
-
- 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
NCT05825131
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- The University of Texas Southwestern Medical Center Dallas, Texas, United States
- Hospices Civils De Lyon Bron, France
Recruiting
MPS (RaDiCo Cohort) (RaDiCo-MPS)
NCT06036693
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +33 more
- Locations
-
- Centre Hospitalier Universitaire d'Angers Angers, France
- Hôpital des Enfants - Groupe Hospitalier Pellegrin Bordeaux, France
- Hôpital Morvan Brest, France
- Hôpital d'Estaing Clermont-Ferrand, France
NCT03333200
- Conditions
- ALD, Alpha-Mannosidosis, Batten Disease, +78 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
NCT06488924
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- Hiroshima University Hospital Hiroshima, Japan
- University of the Ryukyus Hospital Okinawa, Japan
- National Center for Child Health and Development Tokyo, Japan
NCT06567769
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland, California, United States
- University of Minnesota Minneapolis, Minnesota, United States
- National Center for Child Health and Development Setagaya City, Tokyo, Japan
- Ajou University Medical Center Suwon, Gyeongi-do, South Korea
NCT06614894
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +11 more
- Locations
-
- Lysosomal & Rare Disorders Research & Treatment Center, Inc. Fairfax, Virginia, United States
Enrolling by Invitation
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
NCT05368038
- Conditions
- Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +65 more
- Locations
-
- Maimonides Medical Center Brooklyn, New York, United States
- NYU Langone Hospital - Brooklyn Brooklyn, New York, United States
- North Shore University Hospital Manhasset, New York, United States
- NYU Langone Health - Tisch Hospital New York, 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
Enrolling by Invitation
Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE
NCT05492799
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- UPMC Children's Hospital Pittburgh Pittsburgh, Pennsylvania, United States
- Fundación Cardio Infantil - Instituto de Cardiología Bogotá, Colombia
- University Medical Center Hamburg-Eppendorf Hamburg, Germany
Not Yet Recruiting
Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)
NCT07579910
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
Not specified
Not Yet Recruiting
Natural History to Assess Disease in Patients With MPS IIIC
NCT07712003
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +11 more
- Locations
-
- UT Southwestern Children's Medical Center Dallas, Texas, United States
NCT05648851
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- Columbia University Irving Medical Center New York, United States
NCT05523206
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- Sanguine Biosciences, Inc. Woburn, Massachusetts, United States
Completed
Open-label Study of Anakinra in MPS III
NCT04018755
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- The Lundquist Institute at Harbor-UCLA Medical Center Torrance, California, United States
NCT03811028
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- Children´s Hospital and research center Oakland, California, United States
- University of North Carolina hospitals Chapel Hill, North Carolina, United States
- Gazi University Hospital Ankara, Turkey (Türkiye)
NCT03423186
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- Childrens's Hospital and Research Center Oakland, California, United States
- University of North Carolina Hospitals Chapel Hill, North Carolina, United States
- University Medical Center Hamburg-Eppendorf Hamburg, Germany
- Gazi University Hospital Ankara, Turkey (Türkiye)
NCT01155778
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- Emma Children's Hospital, Academic Medical Center Amsterdam, Netherlands
- St. Mary's Hospital Manchester, United Kingdom
NCT01047306
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- University of Minnesota Minneapolis, Minnesota, United States