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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.

12,656 public trials 2,147 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI

NCT01521429

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +8 more
Locations
  • Children's Hospital & Research Center Oakland Oakland, California, United States
  • Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center Torrance, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
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Completed

Streamlined Treatment of Pulmonary Exacerbations in Pediatrics Pilot Study

NCT04608019

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Digestive System Diseases, +5 more
Locations
  • Tucson Cystic Fibrosis Center Tucson, Arizona, United States
  • Children's Hospital of Colorado Aurora, Colorado, United States
  • Children's Healthcare of Atlanta Atlanta, Georgia, United States
  • Lurie Children's Hospital of Chicago & Northwestern University Chicago, Illinois, United States
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Completed

Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840

NCT04422431

Conditions
Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +16 more
Locations
  • Research Site Sacramento, California, United States
  • Research Site Ann Arbor, Michigan, United States
  • Research Site Dallas, Texas, United States
  • Research Site Toronto, Ontario, Canada
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Completed

A Study of KW-3357 in Congenital Antithrombin Deficiency

NCT00938288

Conditions
Antithrombin III Deficiency, Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, +11 more
Locations
  • Department of Haemotogy, Haemastasy, Oncology and Stem Cell Transplantation, Hannover Medical School Hanover, Germany
  • Center for Thrombosis and Haemostasis, Malmo University Hospital Malmö, Sweden
  • Dept Obst Gyn/Section for Women and Child Health/Clinical Trial Unit, Karolinska University Hospital Stockholm, Sweden
  • Treliske, Haematology Clinic, Royal Cornwall Hospital Truro, Cornwall, United Kingdom
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