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Recruiting
A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
NCT04998396
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
- Locations
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- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
- Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND) Boston, Massachusetts, United States
- Weill Cornell Medicine; Division of Pediatric Neurology New York, United States
Recruiting
The Myelin Disorders Biorepository Project
NCT03047369
- Conditions
- 4H Syndrome, ADLD, AGS, +173 more
- Locations
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- 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
Enrolling by Invitation
rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease
NCT04833907
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
- Locations
-
- Dayton Children's Hospital Dayton, Ohio, United States
NCT04126005
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
- Locations
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- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Massachusetts General Hospital Boston, Massachusetts, United States
- University Medical Center Hamburg-Eppendorf Hamburg, Germany
NCT02699190
- Conditions
- 4H Syndrome, ADLD, AGS, +173 more
- Locations
-
- The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
NCT02851563
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
- Locations
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- Massachusetts General Hospital Boston, Massachusetts, United States
- NYU Langone Medical Center New York, United States
- University Medical Center Hamburg-Eppendorf Hamburg, Germany
NCT01999257
- Conditions
- Autonomic Nervous System Diseases, Brain Diseases, Brain Diseases, Metabolic, +33 more
- Locations
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- Montreal General Hospital (MUHC) Montreal, Quebec, Canada
Active, Not Recruiting
Early Check: Expanded Screening in Newborns
NCT03655223
- Conditions
- 17 Alpha-Hydroxylase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, +456 more
- Locations
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- RTI International Research Triangle Park, North Carolina, United States