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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.

12,537 public trials 2,139 recruiting studies
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Genetic Diseases, Inborn trial results

Active, Not Recruiting

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

NCT03913143

Conditions
Blindness, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +26 more
Locations
  • University of Iowa Iowa City, Iowa, United States
  • Universitair Ziekenhuis Gent (UZ) Ghent, Belgium
  • INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte Belo Horizonte, Minas Gerais, Brazil
  • Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP) São Paulo, Brazil
View all 14 locations
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Active, Not Recruiting

An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia

NCT04053803

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of Connecticut Health Center Farmington, Connecticut, United States
  • Foundation for Sickle Cell Disease Research Hollywood, Florida, United States
  • Baylor Scott & White Medical Center - Temple Temple, Texas, United States
  • Bristol Haematology and Oncology Centre Bristol, United Kingdom
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