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Showing 1–18 of 18 trials
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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
-
- AP-HP Hôpital Bicêtre Le Kremlin-Bicêtre, France
- AP-HP Hôpital La Pitié Salpêtrière Paris, France
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
NCT01303146
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- Department of Pediatric Endocrinology and Neurology, Saint Vincent de Paul Hospital Paris, France
NCT03392987
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
- Locations
-
- Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) Milan, Italy
Completed
Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy
NCT01801709
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- Bicêtre Hospital - Paris Sud Le Kremlin-Bicêtre, France
NCT01560182
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
- Locations
-
- Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) Milan, Italy
NCT02699190
- Conditions
- 4H Syndrome, ADLD, AGS, +173 more
- Locations
-
- The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
Completed
Baby Detect : Genomic Newborn Screening
NCT05687474
- Conditions
- 3-Hydroxy 3-Methyl Glutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency, +368 more
- Locations
-
- CRMN, Hôpital La Citadelle Liège, Wallonia, Belgium
NCT04628364
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +23 more
- Locations
-
- National Organization for Rare Disorders Danbury, Connecticut, United States
NCT01586455
- Conditions
- Acute Lymphocytic Leukemia, Acute Myelogenous Leukemia, Adrenal Gland Diseases, +84 more
- Locations
-
- Children's Hospital Colorado Denver, Colorado, United States
- New York Medical College Valhalla, New York, United States
- University of Utah Salt Lake City, Utah, United States
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
NCT00683189
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- Cooper University Hospital Camden, New Jersey, United States
NCT00004378
- Conditions
- Anemia, Anemia, Aplastic, Anemia, Diamond-Blackfan, +47 more
- Locations
-
- University of California Los Angeles Medical Center Los Angeles, California, United States
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
-
- RTI International Research Triangle Park, North Carolina, United States
Active, Not Recruiting
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
NCT02171104
- Conditions
- Abnormalities, Multiple, Acyl-CoA Oxidase Deficiency, Adrenoleukodystrophy With Cerebral Involvement, +85 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
Active, Not Recruiting
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
NCT04283227
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) Milan, Italy