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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.

12,633 public trials 2,161 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

China Post-marketing Surveillance (PMS) Study of Fabrazyme®

NCT05054387

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Investigational Site Number :1560003 Beijing, China
  • Investigational Site Number :1560002 Beijing, China
  • Investigational Site Number :1560001 Shanghai, China
  • Investigational Site Number :1560004 Shanghai, China
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Completed

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

NCT03729362

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • Neuromuscular Research Center Phoenix, Arizona, United States
  • University of Arkansas for Medical Sciences Little Rock, Arkansas, United States
  • University of California, Irvine Irvine, California, United States
  • UF Helath: University of Florida Clinical Research Center Gainesville, Florida, United States
View all 73 locations
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Completed

A Study of BAX 888 in Male Adults With Severe Hemophilia A

NCT03370172

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • Phoenix Childrens Hospital Phoenix, Arizona, United States
  • Orthopaedic Hospital DBA Orthopaedic Hemophilia Treatment Center Los Angeles, California, United States
  • UC Davis Medical Center Sacramento, California, United States
  • University of Colorado Hemophilia & Thrombosis Center Aurora, Colorado, United States
View all 26 locations
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Completed

Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A

NCT04759131

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +6 more
Locations
  • Children's Hospital Los Angeles-Site Number:8400006 Los Angeles, California, United States
  • University of Florida-Site Number:8400009 Gainesville, Florida, United States
  • Children's Healthcare of Atlanta-Site Number:8400019 Atlanta, Georgia, United States
  • Rush University Medical Center-Site Number:8400001 Chicago, Illinois, United States
View all 40 locations
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