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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

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Evaluating Whether Intact Fish Skin Graft Can Decrease the Need for Autograft in the Treatment of Deep Partial-Thickness Burns

NCT07326657

Conditions: Burns, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations: Loyola University Medical Center Maywood, Illinois, United States

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Tissue Sample Study for Mitochondrial Disorders

NCT01803906

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +30 more
Locations: Columbia University New York, United States

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Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease

NCT06880250

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: #13 Astrakhan, Russia

#27 Barnaul, Russia

#7 Borovichi, Russia

#6 Chelyabinsk, Russia

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Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies

NCT04933721

Conditions: Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +15 more
Locations: Study Center Ottawa, Canada

Study Center Brno, Czechia

Study Center Pilsen, Czechia

Study Center Grenoble, France

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Collaborative Approach to Reach Everyone With Familial Hypercholesterolemia (CARE-FH)

NCT05284513

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Familial Hypercholesterolemia, +9 more
Locations: Geisinger Clinic Multiple Locations, Pennsylvania, United States

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A Study Following Males With Haemophilia A on Prophylaxis With Esperoct®

NCT04574076

Conditions: Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations: AKH - Klin. Abt. f. Haematologie u. Haemostaseologie Vienna, Austria

UMHAT "Tsaritsa Yoanna-ISUL" Sofia, Bulgaria

KBC Zagreb, Rebro, Hemofilija centar Zagreb, Croatia

FN Brno odd. hematologie Brno, Czechia

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An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

NCT04049084

Conditions: Adenosine Deaminase Deficiency, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Repair-Deficiency Disorders, +10 more
Locations: Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center Los Angeles, California, United States

UCL Great Ormond Street Institute of Child Health London, United Kingdom

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Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities

NCT05301361

Conditions: Abnormalities, Multiple, Attention Deficit Disorder with Hyperactivity, Attention Deficit Hyperactivity Disorder, +19 more
Locations: UC Davis MIND Institute Sacramento, California, United States

Rush University Chicago, Illinois, United States

Cincinnati Children's Hospital Cincinnati, Ohio, United States

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Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases

NCT01852370

Conditions: Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Chronic Disease, +35 more
Locations: Children's Hospital of Pittsburgh of UPMC Pittsburgh, Pennsylvania, United States

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Mobile App-Based Infection Monitoring in Familial Mediterranean Fever

NCT07212764

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Familial Mediterranean Fever, Familial Mediterranean Fever (FMF ), +5 more
Locations: Istanbul University Faculty of Medicine Istanbul, Fatih, Turkey (Türkiye)

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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