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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.

12,629 public trials 2,161 recruiting studies

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Genetic Diseases, Inborn trial results

Completed

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pociredir

NCT05169580

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations: University of Arkansas for Medical Sciences (UAMS) Little Rock, Arkansas, United States

University of California, Los Angeles Los Angeles, California, United States

Sonar Research Center Atlanta, Georgia, United States

University of Illinois Chicago Chicago, Illinois, United States

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Completed

Study of Olfactory Disorders in Patients With Cardiac Amyloidosis

NCT04661800

Conditions: Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +24 more
Locations: Assistance Publique Hôpitaux de Paris - CHU HENRI MONDOR Créteil, France

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Completed

Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia

NCT04390971

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations: The Affiliated Hospital of Guizhou Medical University Guiyang, Guizhou, China

Affiliated Hospital of Zunyi Medical University Zunyi, Guizhou, China

Institute of Hematology & Blood Diseases Hospital Tianjin, Tianjin Municipality, China

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Completed

In Vitro Effect Study of Interleukin-2 Muteins on Regulatory T Cells of Patients With Different Autoimmune, Allo-immune or Inflammatory Diseases

NCT05544448

Conditions: Acquired Bone Marrow Aplasia, Alopecia, Arthritis, +43 more
Locations: Hôpital Henri Mondor, 1 rue Gustave Eiffel, Créteil, Île-de-France Region, France

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Completed

Psychiatric Manifestations Associated With Behcet's Disease

NCT04585360

Conditions: Anxiety Disorders, Behavior, Behavioral Symptoms, +25 more
Locations: Internal medecine department Le Kremlin-Bicêtre, Val de Marne, France

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Completed

The Montreal Cognitive Assessment.Test in Adults With Sickle Cell Disease

NCT05347043

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +13 more
Locations: Internal Medicine Department - Tenon Paris, France

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Completed

CF And Effects of Drugs Mixed Ex Vivo With Sputum for Mucolytic Treatment

NCT01533636

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations: University of California, San Francisco San Francisco, California, United States

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Completed

Cardiorespiratory and Muscular Rehabilitation of Children and Young Adults With Marfan Syndrome.

NCT03236571

Conditions: Abnormalities, Multiple, Bone Diseases, Bone Diseases, Developmental, +11 more
Locations: CHU de Toulouse Toulouse, Midi-Pyrénées, France

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Completed

A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major

NCT06291961

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations: The First Affiliated Hospital of Guangxi Medical University Nanning, Guangxi, China

Children's Hospital of Fudan University Shanghai, Shanghai Municipality, China

Ruijin Hospital Shanghai JiaoTong University School of Medicine Shanghai, Shanghai Municipality, China

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Completed

Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis

NCT03375047

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations: University of Alabama at Birmingham Birmingham, Alabama, United States

National Jewish Health Denver, Colorado, United States

University of Florida Gainesville, Florida, United States

Northwestern University Chicago, Illinois, United States

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