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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.

12,672 public trials 2,143 recruiting studies

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Genetic Diseases, Inborn trial results

Completed

Pulsatility Index, Vasomotor Reactivity and Leukoencephalopathy in Fabry Patients

NCT04577170

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations: Second Department of Neurology, "Attikon" University Hospital, School of Medicine, National and Kapodistrian University of Athens, Athens, Greece Athens, Greece

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Completed

Examination of Quantitative Electroencephalographic (QEEG) Biomarkers in Huntington's Disease

NCT00670709

Conditions: Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +14 more
Locations: UCLA Laboratory of Brain, Behavior,a nd PHarmacology Los Angeles, California, United States

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Completed

Brief Cognitive Behavioral Therapy to Treat Itch Rumination "Itch CBT" in Eczema

NCT04486742

Conditions: Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations: Lurie Children's Hospital Chicago, Illinois, United States

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Completed

Effects of Social Gaze Training on Brain and Behavior in Fragile X Syndrome

NCT02616796

Conditions: Chromosome Disorders, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +14 more
Locations: Stanford University School of Medicine Stanford, California, United States

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Completed

Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease

NCT05417126

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +6 more
Locations: Nanoscope Clinical Site Miami, Florida, United States

Nanoscope Clinical Site McAllen, Texas, United States

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Completed

Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects

NCT04416126

Conditions: Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations: Auckland Clinical Studies (ACS) Ltd. Auckland, New Zealand

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Completed

Evaluation of Patient Access to People With Cystic Fibrosis (CF) and Healthcare Professionals

NCT06122025

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations: Leeds Teaching Hospitals NHS Trust Leeds, United Kingdom

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Completed

Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases

NCT03939533

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Immune System Diseases, +3 more
Locations: Octapharma Research Site Scottsdale, Arizona, United States

Octapharma Research Site Irvine, California, United States

Octapharma Research Site Santa Barbara, California, United States

Octapharma Research Site Centennial, Colorado, United States

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Completed

A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD

NCT01761292

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Duchenne Muscular Dystrophy (DMD), +12 more
Locations: Azienda Ospedaliera Universitaria Policlinico G. Martino Messina, Italy

IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano Milan, Italy

Policlinico Agostino Gemelli Roma, Italy

Ospedale Pediatrico Bambino Gesù Rome, Italy

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Completed

Pharmacokinetic Study of Oral ALXN1840 in Japanese and Non-Japanese Adult Healthy Participants

NCT05641311

Conditions: Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations: Richmond Pharmacology Ltd., St George's University of London London, United Kingdom

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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