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Genetic Diseases, Inborn

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Genetic Diseases, Inborn

Explore clinical trials studying Genetic Diseases, Inborn. Study availability and eligibility vary by location and protocol.

12,530 public trials 2,140 recruiting studies

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Genetic Diseases, Inborn trial results

Recruiting

Quantitation and Spatial Registration of Airways Dysfunction With Dynamic 19F MRI in Cystic Fibrosis

NCT06057714

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +6 more
Locations: Univeristy of North Carolina at Chapel Hill Chapel Hill, North Carolina, United States

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Safety and Efficacy of Second Infusion of FAP iCDC in End-stage Dilated Cardiomyopathy

NCT07516288

Conditions: Cardiomegaly, Cardiomyopathies, Cardiomyopathy, Dilated, +7 more
Locations: Second Affiliated Hospital, School of Medicine, Zhejiang University Hangzhou, Zhejiang / 浙江, China

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Recruiting

Gastrointestinal Response of Pediatric Cystic Fibrosis Patients on Mediterranean Diet

NCT07223255

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Cystic Fibrosis (CF), +7 more
Locations: Dartmouth Hitchcock Medical Center Lebanon, New Hampshire, United States

Dartmouth Health Children's Manchester, New Hampshire, United States

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Recruiting

A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU)

NCT07672756

Conditions: Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations: The First Affiliated Hospital of Anhui Medical University Hefei, Anhui, China

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Recruiting

Evaluation of Ventilation Defects Downstream of Mucus Plugs in Patients With Muco-Obstructive Lung Disease

NCT06985225

Conditions: Asthma, Bronchial Diseases, COPD, +21 more
Locations: University of Kansas Medical Center Kansas City, Kansas, United States

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Natural History and Genetics of Food Allergy and Related Conditions

NCT02504853

Conditions: Abnormalities, Multiple, Aneurysm, Aortic Aneurysm, +36 more
Locations: National Institutes of Health Clinical Center Bethesda, Maryland, United States

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TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

NCT04356469

Conditions: Anemia, Anemia, Aplastic, Bone Marrow Diseases, +9 more
Locations: Johns Hopkins All Children's Hospital St. Petersburg, Florida, United States

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A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

NCT04064060

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +13 more
Locations: Local Institution - 968 Los Angeles, California, United States

Local Institution - 971 Oakland, California, United States

Local Institution - 978 Stanford, California, United States

Local Institution - 975 Tampa, Florida, United States

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TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children

NCT04414046

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Immune System Diseases, +6 more
Locations: Johns Hopkins All Children's Hospital St. Petersburg, Florida, United States

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Evaluating Efficacy of Tivozanib (AV-951) in Biliary Tract Cancers

NCT04645160

Conditions: Adenocarcinoma, Bile Duct Diseases, Bile Duct Neoplasm, +23 more
Locations: National Institutes of Health Clinical Center Bethesda, Maryland, United States

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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