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NCT01306019
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
- Locations
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- National Institutes of Health Clinical Center Bethesda, Maryland, United States
NCT00128973
- Conditions
- Bronchial Diseases, Bronchiolitis, Bronchiolitis Obliterans, +32 more
- Locations
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- National Institutes of Health Clinical Center Bethesda, Maryland, United States
NCT03217617
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
- Locations
-
- Shenzhen Geno-immune Medical Institute Shenzhen, Guangdong, China
NCT07474935
- Conditions
- Aneurysm, Aneurysm, False, Bifurcation Aneurysms, +28 more
- Locations
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- CHU Brest - Hôpital de La Cavale Blanche Brest, France
- Pitié Salpêtrière Hospital Paris, France
- Helios Klinikum Erfurt Erfurt, Germany
- Universitätsklinikum Hamburg-Eppendorf Hamburg, Germany
NCT03311503
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Gene Therapy, Genetic Diseases, Inborn, +8 more
- Locations
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- Mattel Children's Hospital - UCLA Los Angeles, California, United States
- Emory University/Childrens Healthcare of Atlanta Atlanta, Georgia, United States
- Boston Childrens Hospital Boston, Massachusetts, United States
- Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
NCT03601286
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +7 more
- Locations
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- Great Ormond Street Hospital for Children NHS Foundation Trust London, Greater London, United Kingdom
Enrolling by Invitation
Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy
NCT06851767
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +9 more
- Locations
-
- National Institutes of Health Clinical Center Bethesda, Maryland, United States
Enrolling by Invitation
Natural History Study of SCID Disorders
NCT01186913
- Conditions
- ADA SCID, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Repair-Deficiency Disorders, +17 more
- Locations
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- University of Alabama at Birmingham Birmingham, Alabama, United States
- Phoenix Children's Hospital Phoenix, Arizona, United States
- Children's Hospital Los Angeles Los Angeles, California, United States
- University of California, Los Angeles Los Angeles, California, United States
NCT01129544
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Repair-Deficiency Disorders, Genetic Diseases, Inborn, +9 more
- Locations
-
- Mattel Children's Hospital - UCLA Los Angeles, California, United States
- Children's Hospital Boston Boston, Massachusetts, United States
- Cincinnati Children's Medical Center Cincinnati, Ohio, United States
NCT01410019
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Diseases, X-Linked, +8 more
- Locations
-
- Hopital Necker Paris, France
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
NCT02600364
- Conditions
- Aneurysm, Brain Diseases, Cardiovascular Diseases, +16 more
- Locations
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- Clínica La Sagrada Familia Buenos Aires, Argentina
- Ziekenhuis Oost-Limburg Genk, Belgium
- St. Ivan Rilski Hospital Sofia, Bulgaria
- Groupe Hospitalier Pellegrin Bordeaux, France
NCT00008450
- Conditions
- Adenosine Deaminase Deficiency, Autosomal Recessive Disorder, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +17 more
- Locations
-
- Fred Hutch/University of Washington Cancer Consortium Seattle, Washington, United States
NCT00028236
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Repair-Deficiency Disorders, Genetic Diseases, Inborn, +9 more
- Locations
-
- National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland, United States
NCT00006335
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Repair-Deficiency Disorders, Genetic Diseases, Inborn, +9 more
- Locations
-
- National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland, United States
NCT00113464
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Repair-Deficiency Disorders, Genetic Diseases, Inborn, +9 more
- Locations
-
- Yale University New Haven, Connecticut, 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
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- RTI International Research Triangle Park, North Carolina, United States