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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.

12,584 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

NCT04713475

Conditions
Beta-Galactosidase-1 (GLB1) Deficiency, Brain Diseases, Brain Diseases, Metabolic, +23 more
Locations
  • Benioff Children's Hospital Oakland, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Children's Hospital at St. Peter's University Hospital New Brunswick, New Jersey, United States
  • The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
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Active, not recruiting

Realizing Effectiveness Across Continents With Hydroxyurea (REACH)

NCT01966731

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • Hospital Pediátrico David Bernardino Luanda, Angola
  • Centre Hospitalier Monkole Kinshasa, Democratic Republic of the Congo
  • KEMRI/Wellcome Trust Research Kilifi, Kenya
  • Ministry of Health Mbale Regional Hospital Mbale, Uganda
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Active, not recruiting

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy

NCT06280209

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy, Genetic Diseases, Inborn, +8 more
Locations
  • Fondazione Serena ETS - Centro Clinico NeMO Milano Milan, Italy
  • UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore Rome, Italy
  • Leids Universitair Medisch Centrum Leiden, Netherlands
  • Hospital Sant Joan de Deu Barcelona, Spain
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Active, not recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

NCT05280548

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Alabama -The Kirklin Clinic- Site Number : 8400010 Birmingham, Alabama, United States
  • University of California Los Angeles Medical Center- Site Number : 8400008 Los Angeles, California, United States
  • Emory University School of Medicine - Atlanta- Site Number : 8400009 Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005 Chicago, Illinois, United States
View all 54 locations
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Active, not recruiting

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

NCT04848779

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Advanced Medical Genetics- Site Number : 8400002 Hawthorne, New York, United States
  • Duke University Medical Center- Site Number : 8400004 Durham, North Carolina, United States
  • Cincinnati Children's Hospital Medical Center- Site Number : 8400001 Cincinnati, Ohio, United States
  • Le Bonheur Children's Hospital- Site Number : 8400005 Memphis, Tennessee, United States
View all 15 locations
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