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Recruiting
Krabbe Disease Global Patient Registry
NCT02993796
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
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- State University of New York at Buffalo Buffalo, New York, United States
NCT03725670
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
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- Lung-Ji Chang Shenzhen, Guangdong, China
Recruiting
Adrenoleukodystrophy National Registry Study
NCT03789721
- Conditions
- ALD (Adrenoleukodystrophy), Adrenal Gland Diseases, Adrenal Insufficiency, +23 more
- Locations
-
- Masonic Cancer Center at University of Minnesota Minneapolis, Minnesota, United States
NCT05659901
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
- Locations
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- Ionis Investigative Site Atlanta, Georgia, United States
- Ionis Investigative Site Philadelphia, Pennsylvania, United States
- Ionis Investigative Site Clermont-Ferrand, France
- Ionis Investigative Site Le Kremlin-Bicêtre, France
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
NCT03333200
- Conditions
- ALD, Alpha-Mannosidosis, Batten Disease, +78 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
NCT02714764
- Conditions
- Alexander Disease, Brain Diseases, Brain Diseases, Metabolic, +13 more
- Locations
-
- Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
NCT04528355
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +119 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
NCT07290114
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 more
- Locations
-
- Harran University Research and Application Hospital Şanliurfa, HALİLİYE, Turkey (Türkiye)
NCT01962415
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +125 more
- Locations
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- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
NCT06150716
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
- Locations
-
- Ionis Investigative Site Atlanta, Georgia, United States
- Ionis Investigative Site Philadelphia, Pennsylvania, United States
- Ionis Investigative Site Salt Lake City, Utah, United States
- Ionis Investigative Site Le Kremlin-Bicêtre, France
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
NCT04925349
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +37 more
- Locations
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- AP-HP Hôpital Bicêtre Le Kremlin-Bicêtre, France
- AP-HP Hôpital La Pitié Salpêtrière Paris, France
Recruiting
IT and IV Lentiviral Gene Therapy for X-ALD
NCT03727555
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +22 more
- Locations
-
- Shenzhen Geno-immune Medical Institute Shenzhen, Guangdong, China
NCT02254863
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
-
- Duke University Medical Center Durham, North Carolina, United States
NCT06224413
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +22 more
- Locations
-
- Boston Children's Hospital Boston, Massachusetts, United States
- Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
- UT Southwestern Medical Center Dallas, Texas, United States
NCT07046338
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- Shenzhen Geno-Immune Medical Institute Shenzhen, Guangdong, China
Recruiting
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
NCT01793168
- Conditions
- 1p36 Deletion Syndrome, 3-Methylglutaconic Aciduria, Type V, 4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome, +698 more
- Locations
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- Sanford Health Sioux Falls, South Dakota, United States
- Online Patient Enrollment System Sydney, Australia
NCT05443906
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +33 more
- Locations
-
- Kennedy Krieger Institute Baltimore, Maryland, United States
NCT06796920
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +26 more
- Locations
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- Beijing TianTan Hospital Beijing, Beijing Municipality, China
- The 958 Hospital of Chinese People's Liberation Army, The Jiangbei Campus of Southwest Hospital, The First Affiliated Hospital of Army Medical University Chongqing, Chongqing Municipality, China
- the Southwest hospital Chongqing, Chongqing Municipality, China
- Jingmen No.1 People's Hospital Jingmen, Hubei, China