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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.

12,582 public trials 2,160 recruiting studies

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Genetic Diseases, Inborn trial results

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Optimizing Hydroxyurea Dosage With Pharmakokinetic in Patients Suffering of Moderate to Severe Sickle Cell Anemia

NCT06761560

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations: CHU Sainte-Justine Montreal, Quebec, Canada

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Pilot Study on the Value of Bedside Pleuropulmonary Ultrasound in Patients With Sickle Cell Disease Presenting With Vaso-occlusive Crisis

NCT06755385

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

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A Clinical Study of AK120 in Adolescents With Moderate-to-severe Atopic Dermatitis (AD)

NCT06756126

Conditions: Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations: Dongguan People's Hospital Dongguan, Guangdong, China

The Second Affiliated Hospital of Guangzhou Medical University Guangzhou, Guangdong, China

The University of Hong Kong - Shenzhen Hospital Shenzhen, Guangdong, China

Hunan Pediatric Medical Union Changsha, Hunan, China

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Not Yet Recruiting

Study of QLC7401 in the Treatment of Primary Hypercholesterolemia or Mixed Hyperlipidemia With Elevated LDL Cholesterol

NCT06750341

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Genetic Diseases, Inborn, +9 more
Locations: Not specified

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AI-enabled Screening and Diagnosis of Cardiomyopathies Using Coronary CTA

NCT06748261

Conditions: Amyloid Cardiomyopathy, Amyloid Neuropathies, Amyloid Neuropathies, Familial, +38 more
Locations: Not specified

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KN057 Multiple Dose Study in Patients with Hemophilia a or Hemophilia B with or Without Inhibitors

NCT06747416

Conditions: Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations: Not specified

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Comparison of the Effects of Synchronous and Asynchronous Telerehabilitation in Patients with Juvenile Familial Mediterranean Fever

NCT06743152

Conditions: Behavior, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Exercise, +6 more
Locations: Istanbul University-Cerrahpasa Istanbul, Turkey (Türkiye)

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An Open-label Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of SKP-0141 for the Treatment and Prophylaxis in Severe Hemophilia a Patients

NCT06738901

Conditions: Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations: Not specified

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CBD Delivery with the A-Synaptic GT4 Transdermal Delivery System in with Dravet Syndrome And/or Lennox-Gastaut Syndrome

NCT06738732

Conditions: Brain Diseases, Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +9 more
Locations: Boston Childrens' Hospital Boston, Massachusetts, United States

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Evaluation of Brain Dysfunction in Patients with Duchene Muscular Dystrophy

NCT06732011

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations: Not specified

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