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NCT02714764
- Conditions
- Alexander Disease, Brain Diseases, Brain Diseases, Metabolic, +13 more
- Locations
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- Children's Hospital of Philadelphia Philadelphia, Pennsylvania, 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
Recruiting
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
NCT04880356
- Conditions
- 2-Hydroxyglutaricaciduria, Adrenal Gland Diseases, Adrenal Insufficiency, +58 more
- Locations
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- Fondazione IRCCS Istituto Neurologico Carlo Besta Milan, Milano, Italy
NCT02699190
- Conditions
- 4H Syndrome, ADLD, AGS, +173 more
- Locations
-
- The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
Active, Not Recruiting
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
NCT04849741
- Conditions
- Alexander Disease, Brain Diseases, Brain Diseases, Metabolic, +13 more
- Locations
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- Lucile Packard Children's Hospital Stanford Palo Alto, California, United States
- Children's Hospital of Atlanta Atlanta, Georgia, United States
- Massachusetts General Hospital Boston, Massachusetts, United States
- Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States