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Clinical trial condition

Genetic Diseases, Inborn

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

12,530 public trials 2,140 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Study of Cannabidiol in Sanfilippo Syndrome

NCT06333041

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center Torrance, California, United States
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Recruiting

This Study is a Non-interventional Disease Registry of Adolescent and Adult Patients With Atopic Dermatitis Who Initiate or Switch Any Systemic Treatment

NCT07290803

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +9 more
Locations
  • Cahaba Dermatology & Skin Health Center- Site Number : 8400006 Birmingham, Alabama, United States
  • River Region Dermatology and Laser- Site Number : 8400041 Montgomery, Alabama, United States
  • San Tan Allergy & Asthma- Site Number : 8400031 Gilbert, Arizona, United States
  • Kern Research, Inc.- Site Number : 8400047 Bakersfield, California, United States
View all 59 locations
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Recruiting

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

NCT06891443

Conditions
Blindness, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +28 more
Locations
  • UCSF Wayne and Gladys Valley Center for Vision San Francisco, California, United States
  • University of Miami - Bascom Palmer Eye Institute Miami, Florida, United States
  • University of Iowa Iowa City, Iowa, United States
  • University of Minnesota Medical School Minneapolis, Minnesota, United States
View all 17 locations
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Recruiting

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

NCT04626674

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
  • Stanford University Palo Alto, California, United States
  • University of California, Davis Sacramento, California, United States
  • Washington University in St. Louis St Louis, Missouri, United States
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Recruiting

A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia

NCT05664737

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • Local Institution - 0008 Halifax, Nova Scotia, Canada
  • Sun Yat-sen Memorial Hospital, Sun Yat-Sen University Guangzhou, GD, China
  • Nanfang Hospital of Southern Medical University Guangzhou, GD, China
  • The First People's Hospital of Foshan Foshan, Guangdong, China
View all 36 locations
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Recruiting

TSC Biosample Repository and Natural History Database

NCT05676099

Conditions
Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +23 more
Locations
  • University of Alabama Birmingham Birmingham, Alabama, United States
  • Loma Linda University Children's Hospital Loma Linda, California, United States
  • University of California Los Angeles Los Angeles, California, United States
  • Jack & Julia Center for TSC, Oakland Children's Hospital and Research Center Oakland, California, United States
View all 26 locations
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