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Genetic Diseases, Inborn

Clinical trial condition

Genetic Diseases, Inborn

Explore clinical trials studying Genetic Diseases, Inborn. Study availability and eligibility vary by location and protocol.

456 public trials 107 recruiting studies

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Genetic Diseases, Inborn trial results

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Implementation-effectiveness Trial of Mainstreaming of Clinical Genomic Sequencing for Rare Disease in Ontario, Canada

NCT07799792

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Evaluation of Mainstreamed Models of Genetic Service Delivery, +6 more
Locations: Not specified

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Not yet recruiting

The Genetics Update

NCT07795879

Conditions: Cancer, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, +3 more
Locations: St. Michael's Hospital Toronto, Ontario, Canada

Mount Sinai Hospital, Toronto Toronto, Ontario, Canada

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Not yet recruiting

Use of N-Acetylcysteine in the Treatment of Repetitive and Self-Injurious Behaviors in Cornelia de Lange Syndrome

NCT04381897

Conditions: Abnormalities, Multiple, Behavior, Behavioral Symptoms, +12 more
Locations: Johns Hopkins University School of Medicine Baltimore, Maryland, United States

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Not yet recruiting

The Mayo Clinic Rare and Undiagnosed Disease Hackathon

NCT07714161

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Genetic Diseases, +5 more
Locations: Mayo Clinic Rochester, Minnesota, United States

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Not yet recruiting

Study Evaluating a Gene Therapy for IPEX Syndrome Through the Expression of FOXP3 on Deficient T Cells to Produce Tregs-like.

NCT07697118

Conditions: Autoimmune Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +2 more
Locations: Department of Biotherapy, Hopital Necker Enfants malades Paris, Île-de-France Region, France

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Not yet recruiting

Prenatal Transplantation for Fetuses With Fanconi Anemia

NCT07408583

Conditions: Anemia, Anemia, Aplastic, Anemia, Hypoplastic, Congenital, +13 more
Locations: University of California, San Francisco San Francisco, California, United States

Stanford University Stanford, California, United States

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Multicenter Epidemiological Study for the Evaluation of the Seroprevalence of Total and Neutralizing Antibodies Against Adenoassociated Virus Serotypes Used in Gene Therapy in Patients With Neuromuscular Diseases of Genetic Origin.

NCT07633951

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Genetic Disorder, +1 more
Locations: GENETHON Évry, Île-de-France Region, France

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Not yet recruiting

Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

NCT07473843

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

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Not yet recruiting

BAG3-DCM Natural History Study

NCT07486752

Conditions: Cardiomegaly, Cardiomyopathies, Cardiomyopathy, Dilated, +7 more
Locations: Not specified

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Not yet recruiting

Determinants and Consequences of the Transition to Adulthood for Adolescents With Severe Haemophilia: TRANSHEMO 2, an Ancillary Study to the TRANSHEMO Project

NCT07418827

Conditions: Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations: CHU La Reunion Saint-Denis, La Réunion, France

CH Annecy - St Julien Annecy, France

Chu de Bordeaux Bordeaux, France

Chu de Caen Caen, France

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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