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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.

12,520 public trials 2,145 recruiting studies

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Genetic Diseases, Inborn trial results

Active, Not Recruiting

Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt Disease(STGD1)

NCT06300476

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +6 more
Locations: West China Hospital Chengdu, Sichuan, China

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Physical Abilities of Teenagers With Familial Mediterranean Fever

NCT06257342

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Familial Mediterranean Fever, Genetic Diseases, Inborn, +1 more
Locations: CH de Versailles Le Chesnay, France

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Sociodemographic Factors and Criminal Behaviour Preceding Neurodegenerative Disease - Retrospective Register Study

NCT06209515

Conditions: Alzheimer Disease, Amyotrophic Lateral Sclerosis, Arterial Occlusive Diseases, +53 more
Locations: Kuopio University Hospital Kuopio, Finland

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Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic Mutation

NCT03963154

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +5 more
Locations: •Centre Hospitalier National d'Ophtalmologie (CHNO) des Quinze-Vingts Paris, France

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Stockholm hyperTRIglyceridemia REGister (STRIREG) Study

NCT06104943

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Familial Hypertriglyceridemia, +10 more
Locations: Not specified

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The REPLACE Registry for Cholbam® (Cholic Acid)

NCT03115086

Conditions: Abnormalities, Multiple, Bile Acid Synthesis Disorders, Brain Diseases, +20 more
Locations: University of Alabama Birmingham Birmingham, Alabama, United States

Stanford School of Medicine Palo Alto, California, United States

UC San Francisco San Francisco, California, United States

Kidz Pediatric Multispecialty Group Hollywood, Florida, United States

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Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients

NCT00555555

Conditions: Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Blood Platelet Disorders, +12 more
Locations: Not specified

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Safety, Pharmacokinetics and Efficacy of an ATIII Concentrate

NCT00319228

Conditions: Antithrombin III Deficiency, Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, +10 more
Locations: Not specified

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First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

NCT04798235

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations: Queen's University/Kingston Health Sciences Centre Kingston, Ontario, Canada

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Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)

NCT04687020

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations: UC Davis Sacramento, California, United States

Lurie Children's Hospital Chicago, Illinois, United States

Duke University Medical Center Durham, North Carolina, United States

Children's Hospital of Richmond at VCU Richmond, Virginia, United States

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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