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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.

449 public trials 107 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)

NCT03435627

Conditions
Cardiovascular Abnormalities, Cardiovascular Diseases, Congenital Abnormalities, +11 more
Locations
  • Novo Nordisk Investigational Site Asahikawa, Hokkaido, Japan
  • Novo Nordisk Investigational Site Fukuoka, Japan
  • Novo Nordisk Investigational Site Kanagawa, Japan
  • Novo Nordisk Investigational Site Kanagawa, Japan
View all 22 locations
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Completed

Implementing an Individualized Pain Plan (IPP) for ED Treatment of VOE's in Sickle Cell Disease

NCT04584528

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +8 more
Locations
  • University of California San Francisco Oakland, California, United States
  • Georgia Regents University Augusta, Georgia, United States
  • University of Illinois Chicago, Illinois, United States
  • Washington University St Louis, Missouri, United States
View Trial Details
Completed

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

NCT04201418

Conditions
Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +16 more
Locations
  • Clinical Trial Site Los Angeles, California, United States
  • Clinical Trial Site San Diego, California, United States
  • Clinical Trial Site Aurora, Colorado, United States
  • Clinical Trial Site Farmington, Connecticut, United States
View all 27 locations
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Completed

Natural History Study of CEP290-Related Retinal Degeneration

NCT03396042

Conditions
Blindness, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +17 more
Locations
  • Bascom Palmer Eye Institute Miami, Florida, United States
  • Massachusetts Eye and Ear Infirmary Boston, Massachusetts, United States
  • W.K. Kellogg Eye Center Ann Arbor, Michigan, United States
  • Casey Eye Institute - OHSU Portland, Oregon, United States
View Trial Details
Completed

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

NCT03417245

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
Locations
  • Investigational Site Number 0140 Little Rock, Arkansas, United States
  • Investigational Site Number 128 Gainesville, Florida, United States
  • Investigational Site Number 103 Tampa, Florida, United States
  • Investigational Site Number 102 Chicago, Illinois, United States
View all 64 locations
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