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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.

449 public trials 107 recruiting studies
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Genetic Diseases, Inborn trial results

Enrolling by Invitation

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

NCT07393373

Conditions
Bone Diseases, Bone Diseases, Developmental, Carbohydrate Metabolism, Inborn Errors, +16 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Childrens Hospital Colorado Aurora, Colorado, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
  • Johns Hopkins School of Medicine Baltimore, Maryland, United States
View all 24 locations
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Enrolling by Invitation

A Long-term Follow-up Study in Participants Who Received CTX001

NCT04208529

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +11 more
Locations
  • Lucile Packard Children's Hospital Palo Alto, California, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology Chicago, Illinois, United States
  • Herbert Irving Pavilion - Hematology New York, United States
  • New York Presbyterian Hospital - Morgan Stanley Children's Hospital New York, United States
View all 20 locations
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Enrolling by Invitation

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

NCT05768386

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Cardiovascular Diseases, +9 more
Locations
  • Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center Los Angeles, California, United States
  • UC Davis Hemophilia Treatment Center Sacramento, California, United States
  • University of California San Diego, Hemophilia & Thrombosis Treatment Center San Diego, California, United States
  • University of California San Francisco - Moffit Hospital, Outpatient Hematology Clinic San Francisco, California, United States
View all 41 locations
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Enrolling by Invitation

Extension Study of Infigratinib in Children With Achondroplasia (ACH)

NCT05145010

Conditions
Achondroplasia, Bone Diseases, Bone Diseases, Developmental, +15 more
Locations
  • USCF Benioff Children's Hospital, Oakland Oakland, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Nemours Alfred I. Dupont Hospital for Children Wilmington, Delaware, United States
  • Johns Hopkins University Baltimore, Maryland, United States
View all 31 locations
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Enrolling by Invitation

SLC13A5 Deficiency Natural History Study - United States Only

NCT06144957

Conditions
Brain Diseases, Central Nervous System Diseases, Citrate Transporter Deficiency, +16 more
Locations
  • Lucille Packard Children's Hospital, Stanford University Palo Alto, California, United States
  • Brown University Providence, Rhode Island, United States
  • University of Texas Southwestern Dallas Dallas, Texas, United States
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Enrolling by Invitation

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

NCT06363760

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +10 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Smilow Cancer Hospital New Haven, Connecticut, United States
  • Johns Hopkins All Children's Hospital St. Petersburg, Florida, United States
View all 18 locations
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