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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.

12,656 public trials 2,147 recruiting studies

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Genetic Diseases, Inborn trial results

Completed

Study of in Vivo and in Vitro Transcriptomic and Proteomic Signatures in Unhereditary Ichtyosis

NCT05312073

Conditions: Autosomal Recessive Congenital Ichthyosis, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +11 more
Locations: Saint Louis Hospital Paris, France

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Completed

Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia

NCT04233918

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Genetic Diseases, Inborn, +9 more
Locations: Regeneron Research Site Wilmington, Delaware, United States

Regeneron Research Site Boca Raton, Florida, United States

Regeneron Research Site Kansas City, Kansas, United States

Regeneron Research Center Boston, Massachusetts, United States

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Completed

Effects of Calorie Restriction and Cold Stimuli on Health-related Indicators, Cognitive and Motor Functions

NCT05545943

Conditions: Behavior, Body Weight, Body Weight Changes, +21 more
Locations: Lithuanian Sports University Kaunas, Lithuania

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Completed

Promoting Health Through Parent Empowerment and the Activation of Routines (PrO-PEAR)

NCT05020366

Conditions: Abnormalities, Multiple, Behavior, Chromosome Disorders, +9 more
Locations: Down Syndrome Center of Western Pennsylvania Pittsburgh, Pennsylvania, United States

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Completed

Contractile Cross Sectional Areas and Muscle Strength in Patients With Congenital Myopathies

NCT03018184

Conditions: Congenital Myopathy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +10 more
Locations: Copenhagen Neuromuscular Center, Rigshospitalet Copenhagen, Denmark

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Completed

Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A

NCT05265767

Conditions: Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations: Christian Medical College Vellore Ranipet Campus Vellore, Tamil Nadu, India

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Completed

An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment

NCT04165135

Conditions: Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +9 more
Locations: AOU Cons. Policlinico Bari; Centro Emofilia e Trombosi - UO Medicina Trasfusionale Bari, Apulia, Italy

Az. Osp. Pugliese Ciaccio; S. C. Emofilia, Emostasi e Trombosi Catanzaro, Calabria, Italy

AOU Federico II; Medicina Clinica Chirurgia Centro Emocoaugulopatie e Emofilia Naples, Campania, Italy

PO Cesena - Osp M. Bufalini; Patologia Clinica - S.S.A. Serv. Medicina Trasf.- Centro Emofilia Cesena, Emilia-Romagna, Italy

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Completed

Treatment of Transcranial Alternating Current Stimulation（tACS）on Cerebellar Ataxia

NCT05557786

Conditions: Ataxia, Brain Diseases, Central Nervous System Diseases, +15 more
Locations: Department of Neurology, The First Affiliated Hospital Fujian Medical University Fuzhou, Fujian, China

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Completed

Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)

NCT04973280

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +10 more
Locations: Local Institutions Vienna, Austria

Local Institutions Brussels, Belgium

Local Institutions Berlin, Germany

Local Institutions Rome, Italy

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Completed

Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders

NCT00947544

Conditions: Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +13 more
Locations: UCLA Los Angeles, California, United States

The George Washington DC Children's National Medical Center Washington D.C., District of Columbia, United States

Mount Sinai School of Medicine New York, United States

University of Pittsburgh Pittsburgh, Pennsylvania, United States

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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