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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
NCT02254863
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
-
- Duke University Medical Center Durham, North Carolina, United States
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
NCT04470713
- Conditions
- AB Variant Gangliosidosis GM2, Brain Diseases, Brain Diseases, Metabolic, +24 more
- Locations
-
- Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
- Mayo Clinic - Rochester Rochester, Minnesota, United States
- Lysosomal and Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
- UCL Cliniques Universitaires Saint-Luc Brussels, Belgium
NCT00383448
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
NCT00176904
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
NCT01626092
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +50 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
NCT00418847
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
- The Hospital for Sick Children Toronto, Ontario, Canada
Completed
Gene Therapy for Tay-Sachs Disease
NCT01869270
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- Data Management and Coordinating Center (DMCC), Univ. of South Florida Tampa, Florida, United States
- University of Minnesota Minneapolis, Minnesota, United States
Completed
Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
NCT01102686
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
- Locations
-
- The Hospital for Sick Children Toronto, Ontario, Canada
NCT00672022
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
- Children's National Medical Center Washington D.C., District of Columbia, United States
Active, Not Recruiting
Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
NCT06614569
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- UMass Chan Medical School Worcester, Massachusetts, United States
Active, Not Recruiting
First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
NCT04798235
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
- Queen's University/Kingston Health Sciences Centre Kingston, Ontario, Canada