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NCT07054515
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 more
- Locations
-
- UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
- University of Minnesota Medical School Minneapolis, Minnesota, United States
- Mayo Clinic Childrens Center - PIN Rochester, Minnesota, United States
- Children's Medical Center Dallas Dallas, Texas, United States
NCT00029965
- Conditions
- Bone Diseases, Bone Diseases, Metabolic, Brain Atrophy, +29 more
- Locations
-
- National Institutes of Health Clinical Center Bethesda, Maryland, United States
NCT07399704
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +23 more
- Locations
-
- Associação Hospitalar de Prot à Infância Dr. Raul Carneiro Água Verde, Curitiba, Brazil
- Hospital de Clinicas de Porto Alegre Porto Alegre, Rio Grande do Sul, Brazil
- Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira Rio de Janeiro, Brazil
NCT00668187
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +22 more
- Locations
-
- University of Minnesota - Pediatric Genetics and Metabolism Minneapolis, Minnesota, United States
NCT03333200
- Conditions
- ALD, Alpha-Mannosidosis, Batten Disease, +78 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
NCT03952637
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
- Locations
-
- National Institutes of Health Clinical Center Bethesda, Maryland, United States
Recruiting
The Myelin Disorders Biorepository Project
NCT03047369
- Conditions
- 4H Syndrome, ADLD, AGS, +173 more
- Locations
-
- Children's Hospital of Los Angeles Los Angeles, California, United States
- Children's Hospital of Orange County Orange, California, United States
- Stanford University (Lucile Packard Children's Hospital) Palo Alto, California, United States
- University of California, Davis (UC Davis Health) Sacramento, California, United States
NCT02254863
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
-
- Duke University Medical Center Durham, North Carolina, United States
NCT06539169
- Conditions
- Alpha-Thalassemia, Alzheimer Disease, Amyloidosis, +104 more
- Locations
-
- xCures Los Altos, California, 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
Not Yet Recruiting
Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial
NCT07479953
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
Not specified
Not Yet Recruiting
Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis
NCT07445490
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- Département de Neurologie Paris, France
NCT05758922
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +22 more
- Locations
-
- Hospital Pequeno Principe Curitiba, Brazil
- Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
- Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira Rio de Janeiro, Brazil
NCT02851862
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- Massachusetts General Hospital Boston, Massachusetts, United States
NCT02699190
- Conditions
- 4H Syndrome, ADLD, AGS, +173 more
- Locations
-
- The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
NCT04041102
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
- Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil
- Montreal Children's Hospital Research Institute - McGill University Montreal, Quebec, Canada
NCT05109793
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland, California, United States
- Mayo Clinic Rochester Rochester, Minnesota, United States
- Hospital Pequeno Principe Curitiba, Brazil
- Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
NCT03759665
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
- University of California - Los Angeles Los Angeles, California, United States
- Mayo Clinic Rochester, Minnesota, United States
- NYU Langone School of Medicine New York, United States
- University of Giessen Giessen, Germany
NCT04084301
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +32 more
- Locations
-
- Lukas Lannemyr Gothenburg, Västra Götaland County, Sweden
NCT04310163
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- Casimir Trials Plymouth, Massachusetts, United States