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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.

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Genetic Diseases, Inborn trial results

Enrolling by Invitation

Clean Air for Rare MUcociliary Clearance dIsorders

NCT07417267

Conditions: Abnormalities, Multiple, Ciliary Motility Disorders, Ciliopathies, +12 more
Locations: Medical School, University of Cyprus Nicosia, Aglantzia, Cyprus

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Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV

NCT07398872

Conditions: Bone Diseases, Bone Diseases, Metabolic, Brain Diseases, +15 more
Locations: Children's Hospital Zhejiang University School of Medicine Hangzhou, Zhejiang, China

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A Long-term Follow-up Study in Participants Who Received CS-101

NCT06685536

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations: The First Affiliated Hospital of Guangxi Medical University Nanning, Guangxi, China

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A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection

NCT06717932

Conditions: Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations: The First Affiliated Hospital of Guangxi Medical University Nanning, China

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CorEvitas Atopic Dermatitis Registry: A Study of Post Approval Drug Safety and Effectiveness

NCT04504955

Conditions: Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations: CorEvitas, LLC Waltham, Massachusetts, United States

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Penn Family Screening for Familial Hypercholesterolemia

NCT05750667

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Familial Hypercholesterolemia, +9 more
Locations: University of Pennsylvania, Perelman School of Medicine Philadelphia, Pennsylvania, United States

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A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

NCT05967351

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations: Arkansas Children's Hospital Little Rock, Arkansas, United States

UCLA Medical Center Los Angeles, California, United States

Lucile Packard Children's Hospital Stanford (LPCH) Palo Alto, California, United States

University of California, Davis Sacramento, California, United States

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Thera-Clean® Microbubbles System in Patients With Skin Diseases

NCT07050810

Conditions: Atopic Dermatitis, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +18 more
Locations: Northwestern University Department of Dermatology Chicago, Illinois, United States

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Strategy for Management of Patients With Hereditary Cancer Syndromes (HCS) in a Rural Environment

NCT07381985

Conditions: BRCA1 Hereditary Breast and Ovarian Cancer Syndrome, Colonic Diseases, Colorectal Neoplasms, +17 more
Locations: University of Vermont Medical Center Burlington, Vermont, United States

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Palliative Care Needs of Children With Rare Diseases and Their Families

NCT06938542

Conditions: Abnormalities, Multiple, Anxiety Disorders, Arthrogryposis Congenita Multiplex With Intestinal Atresia, +84 more
Locations: Children's National Hospital Washington D.C., District of Columbia, United States

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Genetic Diseases, Inborn

Genetic Diseases, Inborn trial results

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