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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.

12,629 public trials 2,161 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

NCT04138277

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of Arkansas for Medical Sciences Little Rock, Arkansas, United States
  • University of California, Irvine Irvine, California, United States
  • University of Florida Clinical Research Center Gainesville, Florida, United States
  • Emory Clinic Atlanta, Georgia, United States
View all 60 locations
View Trial Details
Completed

Study of Immune Tolerance and Capacity for Wound Healing of Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)

NCT01874769

Conditions
Collagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations
  • Service de dermatologie Necker Hospital for sick children Paris, France
  • Inserm U781 Service de Génétique Necker Hospital for sick children Paris, France
  • Guy's and ST Thomas NHS Foundation trust/Guy's Hospital London, United Kingdom
View Trial Details
Completed

A Study of NIDO-361 in Patients With SBMA

NCT06411912

Conditions
Bulbo-Spinal Atrophy, X-Linked, Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +11 more
Locations
  • Rigshospitalet, Klinik for Nerve- og Muskelsygdomme & Copenhagen Neuromuscular Center Copenhagen, Denmark
  • IRCCS Istituto Neurologico Carlo Besta Milan, Italy
  • Azienda Ospedale Università di Padova Padova, Italy
  • Kyungpook National University Chilgok Hospital Daegu, South Korea
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Completed

Hybrid Effectiveness-Implementation Trial of Guided Relaxation and Acupuncture for Chronic Sickle Cell Disease Pain

NCT04906447

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +7 more
Locations
  • University of Florida Gainesville, Florida, United States
  • Emory University Atlanta, Georgia, United States
  • University of Illinois at Chicago Chicago, Illinois, United States
  • Johns Hopkins University Baltimore, Maryland, United States
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