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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.

12,559 public trials 2,153 recruiting studies

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Genetic Diseases, Inborn trial results

Recruiting

Angelman Syndrome Natural History Study

NCT04507997

Conditions: Abnormalities, Multiple, Angelman Syndrome, Central Nervous System Diseases, +7 more
Locations: Cedars-Sinai Guerin Children's Los Angeles, California, United States

Rady Children's Hospital, San Diego San Diego, California, United States

Children's Hospital Colorado, Anschutz Medical Campus Aurora, Colorado, United States

Emory University School of Medicine Atlanta, Georgia, United States

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Recruiting

Multimodal Electrophysiological Study of Cortico-subcortical Biomarkers of Tics in Tourette Syndrome

NCT06909656

Conditions: Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +10 more
Locations: CHU de Bordeaux Bordeaux, France

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Recruiting

Efficacy Anda Safety of Etoricoxib With Betamethasone for the Treatment of Acute Gout Arthritis

NCT06863701

Conditions: Arthritis, Arthritis, Gouty, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +12 more
Locations: Laboratorio Silanes, S.A. de C.V. Mexico City, Mexico

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Recruiting

BCMA-CD19 CAR-T Therapy for Refractory Autoimmune Diseases

NCT06794008

Conditions: ANCA-associated Vasculitis, Acquired Thrombotic Thrombocytopenic Purpura, Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis, +62 more
Locations: Department of Rheumatology and Immunology, Peking University People's Hospital Beijing, Beijing Municipality, China

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Recruiting

Description of a Retrospective Cohort of Patients With Transthyretin Cardiac Amyloidosis (ATTR-CA) in Internal Medicine

NCT06907836

Conditions: Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +13 more
Locations: Service de Médecine Interne - CHU de Strasbourg - France Strasbourg, France

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Recruiting

Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy

NCT06900049

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy (DMD), Genetic Diseases, Inborn, +8 more
Locations: Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine Shanghai, Shanghai Municipality, China

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Recruiting

Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome

NCT05789056

Conditions: Abnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations: Site #1 San Diego, California, United States

Site #4 Indianapolis, Indiana, United States

Site #5 Quincy, Massachusetts, United States

Site #2 San Antonio, Texas, United States

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Recruiting

Pilot Study "AHSP as a Biomarker of Sickle Cell Disease in a Population of Adults and Children"

NCT06735625

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations: Centre Hospitalier de Saint-Denis Saint-Denis, France

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Recruiting

Transient Pseudohypoaldosteronism Affecting Children With Urinary Tract Malformation

NCT06905600

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, +7 more
Locations: Service pédiatrie 1 - CHU de Strasbourg - France Strasbourg, France

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Recruiting

Pressure Pain Tolerance in Relation to Balance and Strength in Children

NCT06529874

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations: El-Tahrir st.- in front of Ben El-Sarayat, Ad Doqi Al Giza, Giza Governate Giza, Egypt

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Genetic Diseases, Inborn

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