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NCT05682144
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +11 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- University of Minnesota Minneapolis, Minnesota, United States
NCT05619900
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +36 more
- Locations
-
- University of California San Francisco San Francisco, California, United States
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
NCT04528355
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +119 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
NCT01962415
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +125 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
NCT06519552
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- West China Hospital, Sichuan Universit Chengdu, Sichuan, China
Enrolling by Invitation
Long Term Follow-Up for RGX-111
NCT06103487
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- Children's Hospital of Orange County Orange, California, United States
- Hospital de Clinicas de Porto Alegre Porto Alegre, Rio Grande do Sul, Brazil
Completed
Mucopolysaccharidosis I (MPS I) Registry
NCT00144794
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- Phoenix Children's Hospital- Site Number : 840003 Phoenix, Arizona, United States
- Arkansas Children's Hospital- Site Number : 840109 Little Rock, Arkansas, United States
- Southern California Permanente Medical Group- Site Number : 840108 Los Angeles, California, United States
- USC Health Sciences Center Dept of Genetics- Site Number : 840082 Los Angeles, California, United States
NCT05134571
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- Investigational Site Number :1560003 Beijing, China
- Investigational Site Number :1560002 Beijing, China
- Investigational Site Number :1560004 Guangzhou, China
- Investigational Site Number :1560006 Hangzhou, China
Completed
An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)
NCT04453085
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
- Instituto de Genética e Erros Inatos do Metabolismo - IGEIM São Paulo, Brazil
- Fukuoka Children's Hospital Fukuoka, Japan
Completed
Baby Detect : Genomic Newborn Screening
NCT05687474
- Conditions
- 3-Hydroxy 3-Methyl Glutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency, +368 more
- Locations
-
- CRMN, Hôpital La Citadelle Liège, Wallonia, Belgium
NCT04227600
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
- Instituto de Genética e Erros Inatos do Metabolismo - IGEIM São Paulo, Brazil
- Fukuoka Children's Hospital Fukuoka, Japan
NCT00695279
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +31 more
- Locations
-
- St. Jude Children's Research Hospital Memphis, Tennessee, United States
Completed
Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients
NCT06406153
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- Clinical Research Development Unit of Akbar Hospital, Faculty of Medicine Mashhad, Iran
- Growth and Development Research Center, Childrens Medical Center Tehran, Iran
- Loghman Hospital Tehran, Iran
- Mofid Childrens Hospital Tehran, Iran
NCT05073783
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- Investigational Site Italy Italy
NCT03513328
- Conditions
- Acquired Anemia Hemolytic, Acquired Neutropenia in Newborn, Acquired Thrombocytopenia, +82 more
- Locations
-
- UF Health Shands Children's Hospital Gainesville, Florida, United States
NCT03053089
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- HCPA - Hospital das Clinicas de Porto Alegre Porto Alegre, Rio Grande do Sul, Brazil
Completed
Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
NCT03071341
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- HCPA - Hospital das Clinicas de Porto Alegre Porto Alegre, Rio Grande do Sul, Brazil
Completed
Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I
NCT02371226
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
- Locations
-
- Children's Hospital Oakland Oakland, California, United States
- Children's Hospital of Orange County Orange, California, United States
- Emory Healthcare Decatur, Georgia, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
NCT01586455
- Conditions
- Acute Lymphocytic Leukemia, Acute Myelogenous Leukemia, Adrenal Gland Diseases, +84 more
- Locations
-
- Children's Hospital Colorado Denver, Colorado, United States
- New York Medical College Valhalla, New York, United States
- University of Utah Salt Lake City, Utah, United States