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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

GenSci144 Tablets Phase I Clinical Trial

NCT07685210

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • The First Affiliated Hospital of Shandong First Medical University (Shandong Qianfoshan Hospital). Jinan, Shandong, China
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Recruiting

Study of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein (FRSW107) as Prophylactic Treatment.

NCT07684898

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College Tianjin, Tianjin Municipality, China
  • Fuyang Hospital, Affiliated to Anhui Medical University Fuyang, China
  • Fujian Medical University Union Hospital Fuzhou, China
  • Nanfang Hospital of Southern Medical University Guangzhou, China
View all 19 locations
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Recruiting

A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease

NCT06481306

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of Alabama at Birmingham Birmingham, Alabama, United States
  • University of California San Diego - La Jolla La Jolla, California, United States
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Yale-New Haven Hospital New Haven, Connecticut, United States
View all 33 locations
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Recruiting

TIDES 2.0: Prevalence and Longitudinal Course of Depression, Anxiety, and Behavior Problems in Children With Cystic Fibrosis Under 12 Years of Age

NCT07048574

Conditions
Anxiety Disorders, Behavior, Behavioral Symptoms, +11 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Joe DiMaggio Hollywood, Florida, United States
  • Nemours Foundation Orlando, Florida, United States
View all 13 locations
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Recruiting

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

NCT06636383

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +8 more
Locations
  • Children's Hospital of Orange County Orange, California, United States
  • Children's Hospital Colorado Denver, Colorado, United States
  • University of Connecticut Health Center Hartford, Connecticut, United States
  • University of Michigan Ann Arbor, Michigan, United States
View all 19 locations
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Recruiting

A Study to Evaluate the Efficacy and Safety of Subcutaneous Amlitelimab on Background Topical Corticosteroids Therapy in Participants Aged 12 Years and Older With Moderate-to-severe AD Who Have Had an Inadequate Response to Prior Biologic Therapy or an Oral JAK Inhibitor

NCT06241118

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, Dermatitis Atopic, +9 more
Locations
  • University of Alabama at Birmingham- Site Number : 8401267 Birmingham, Alabama, United States
  • Center for Dermatology and Plastic Surgery- Site Number : 8401119 Scottsdale, Arizona, United States
  • Arkansas Dermatology - North Little Rock- Site Number : 8401244 North Little Rock, Arkansas, United States
  • Encino Research Center- Site Number : 8401042 Encino, California, United States
View all 150 locations
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Recruiting

Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

NCT04819841

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • Children's Hospital Los Angeles Los Angeles, California, United States
  • Lucile Packard Children's Hospital Palo Alto, California, United States
  • Washington University St Louis, Missouri, United States
  • Columbia University Irving Medical Center New York, United States
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