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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.

12,633 public trials 2,161 recruiting studies
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Genetic Diseases, Inborn trial results

Completed

Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases

NCT02349906

Conditions
Bone Marrow Diseases, Bone Marrow Failure Disorders, Bone Marrow Failure Syndromes, +11 more
Locations
  • University Hospital Motol, Dep. of Paediatric Haematology and Oncology Prague, Czechia
  • Department of Pediatric Oncology & Hematology, Charite Berlin Berlin, Germany
  • University Children's Hospital Essen Pediatric stem cell transplantation Essen, Germany
  • University Hospital Frankfurt Frankfurt am Main, Germany
View all 21 locations
View Trial Details
Completed

Repository for Inherited Eye Diseases

NCT00378742

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +7 more
Locations
  • National Institutes of Health Clinical Center Bethesda, Maryland, United States
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Completed

A Study to Investigate the Effectiveness and Safety of Bempedoic Acid in Patients With Primary Hypercholesterolemia or Mixed Dyslipidemia in Taiwan

NCT06925100

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Genetic Diseases, Inborn, +9 more
Locations
  • Kaohsiung Medical University Chung-Ho Memorial Hospital Kaohsiung City, Taiwan
  • Far Eastern Memorial Hospital New Taipei City, Taiwan
  • Linkou Chang Gung Memorial Hospital Taoyuan, Taiwan
View Trial Details
Completed

A Research Study Looking at How Safe it is to Switch From Emicizumab to Mim8 in People With Haemophilia A (FRONTIER 5)

NCT05878938

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
Locations
  • Children's Hospital Los Angeles - Endocrinology Los Angeles, California, United States
  • UC Davis Medical Center Sacramento, California, United States
  • UC Denver Hemoph & Thrombo Ctr Aurora, Colorado, United States
  • St Joseph's Hospital Foundation Tampa, Florida, United States
View all 36 locations
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Completed

A Study to Evaluate the Safety, Tolerability, Drug Levels, and Drug Effects of BMS-986326 in Participants With Atopic Dermatitis

NCT06248814

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, Dermatitis, Atopic, +9 more
Locations
  • Local Institution - 0004 Prague, PR, Czechia
  • Local Institution - 0009 Pardubice, Czechia
  • Local Institution - 0006 Clermont-Ferrand, France
  • Local Institution - 0010 Nice, France
View all 13 locations
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Completed

Factors Associated With Hypoventilation in the Myotonic Dystrophy, Progressive Profile Over 5 Years

NCT03764150

Conditions
Apnea, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyssomnias, +17 more
Locations
  • Centre Hospitalier Regional D' Angers Angers, France
  • Ap-Hp Hu Paris Site Raymond Poincare Garches, France
  • Hôpital Calmette, CHRU Lille, France
  • Chu Montpellier Montpellier, France
View Trial Details
Completed

Evaluation of a New Strategy for the Diagnosis of Peroxisomal Diseases

NCT03163771

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diagnoses Disease, Genetic Diseases, Inborn, +4 more
Locations
  • Département de Pédiatrie, Unité de Génétique Clinique, CHU d'Amiens Amiens, France
  • Pédiatrie, CHU Clémenceau de Caen Caen, France
  • Hôpital Jeanne de Flandres, CHRU Lille, France
  • Pédiatrie, Pavillon Mère et Enfant, CHU Ch. Nicolle de Rouen Rouen, France
View Trial Details

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