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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.

12,578 public trials 2,158 recruiting studies

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Genetic Diseases, Inborn trial results

Not Yet Recruiting

iBCI Optimization for Veterans With Paralysis

NCT05470478

Conditions: Amyotrophic Lateral Sclerosis, Brain Diseases, Brain Infarction, +38 more
Locations: Providence VA Medical Center, Providence, RI Providence, Rhode Island, United States

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Haloperidol For Outpatient Symptom Management in Patients Diagnosed With Cannabinoid Hyperemesis Syndrome in the Emergrency Department

NCT07726069

Conditions: Albinism, Cannabinoid Hyperemesis Syndrome, Chediak-Higashi Syndrome, +15 more
Locations: University of Illinois Hospital Chicago, Illinois, United States

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CD19-B Cell Depletion in PAIS-ME/CFS Patients

NCT07724834

Conditions: Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, COVID-19, +37 more
Locations: Charité - Universitätsmedizin Berlin Berlin, Germany

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A Dose-ranging Study of TTI-0102 in Adults and Children With Leigh Syndrome Spectrum (LSS)

NCT06990984

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations: Not specified

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Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia

NCT07721480

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations: Not specified

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CPAP for Hypoxemic Acute Chest Syndrome in Sickle Cell Disease

NCT07703566

Conditions: Acute Chest Syndrome, Acute Chest Syndrome (ACS), Anemia, +12 more
Locations: Not specified

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A Phase III Study of KHN939 in Chinese Patients With Moderate-to-Severe Active Thyroid Eye Disease

NCT07720635

Conditions: Autoimmune Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Endocrine System Diseases, +12 more
Locations: Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University Shanghai, Shanghai Municipality, China

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Bempedoic Acid Therapy for Polycystic Kidney Disease

NCT07282821

Conditions: ADPKD (Autosomal Dominant Polycystic Kidney Disease), Abnormalities, Multiple, Ciliopathies, +12 more
Locations: University of Maryland, Baltimore Baltimore, Maryland, United States

Tufts University Medical Center Boston, Massachusetts, United States

University of Vermont Medical Center Burlington, Vermont, United States

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A Phase III Study of KHN939 in Chinese Patients With Moderate-to-Severe Inactive Thyroid Eye Disease

NCT07720648

Conditions: Autoimmune Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Endocrine System Diseases, +12 more
Locations: Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University Shanghai, Shanghai Municipality, China

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NAD Augmentation to Prevent or Reverse Alzheimer's Disease in People With Down Syndrome

NCT07278492

Conditions: Abnormalities, Multiple, Chromosome Disorders, Congenital Abnormalities, +8 more
Locations: Massachusetts General Hospital Boston, Massachusetts, United States

Brigham and Women's Hospital Boston, Massachusetts, United States

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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