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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.

449 public trials 107 recruiting studies

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Genetic Diseases, Inborn trial results

Recruiting

UW Undiagnosed Genetic Diseases Program

NCT04586075

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Genetic Disease, +6 more
Locations: University of Wisconsin School of Medicine and Public Health Madison, Wisconsin, United States

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Recruiting

A Long-Term Observational Study of Patients With Fucosidosis

NCT07615400

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +11 more
Locations: Children's Hospital of Orange County Orange, California, United States

University of Minnesota Health Minneapolis, Minnesota, United States

Indira Gandhi Institute of Child Health, Department of Pediatric Neurology Bengaluru, India

Erasmus University Medical Center Rotterdam, Netherlands

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Recruiting

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

NCT07608432

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DMD, Duchenne Muscular Dystrophy (DMD), +21 more
Locations: Rare Disease Research, LLC Hillsborough, North Carolina, United States

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Recruiting

Using a Speech-Generating Device to Support Communication in Rare Genetic Conditions

NCT07039084

Conditions: Augmentative and Alternative Communication, Behavior, Communication, +6 more
Locations: Murdoch Children's Research Institute Melbourne, Victoria, Australia

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Recruiting

Developing Protocols for Modelling of Genetic Diseases Using Induced Pluripotent Stem Cells

NCT03612310

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Disease, Genetic Diseases, Inborn
Locations: Alba Science Edinburgh, Midlothian, United Kingdom

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Recruiting

Natural History of Type 1 Interferonopathies: Insights From a European Cohort

NCT07040774

Conditions: Autoimmune Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Disease, +5 more
Locations: Medical University Innsbruck Innsbruck, Austria

Antwerp University Hospital Antwerp, Belgium

Children's Hospital Zagreb Zagreb, Croatia

Motol University Hospital Prague, Czechia

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Recruiting

Genetic Disorders of Obesity Program Database

NCT05747976

Conditions: Body Weight, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Disease, +10 more
Locations: Baylor College of Medicine Houston, Texas, United States

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Recruiting

Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)

NCT07224360

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations: UAMS Medical Center Little Rock, Arkansas, United States

The Foundation for Sickle Cell Disease Hollywood, Florida, United States

Center for Cancer and Blood Disorders Bethesda, Maryland, United States

Southern Specialty Research Flowood, Mississippi, United States

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Recruiting

BCI@Home: Brain Computer Interface Solutions to Enable Youth Living With Severe Disabilities

NCT07073378

Conditions: Acquired Brain Injury (Including Stroke), Brain Damage, Chronic, Brain Diseases, +11 more
Locations: Alberta Children's Hospital Calgary, Alberta, Canada

Glenrose Rehabilitation Hospital Edmonton, Alberta, Canada

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Recruiting

I-TOPS With Patients With Genetic Syndrome SYN-TOPS

NCT07563218

Conditions: Behavior, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +3 more
Locations: Scientific Institute, IRCCS E. Medea, Bosisio Parini, Lecco, Italy Bosisio Parini, Lecco, Italy

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