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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.

12,573 public trials 2,159 recruiting studies
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Genetic Diseases, Inborn trial results

Enrolling by Invitation

Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome

NCT07450664

Conditions
Abnormalities, Multiple, Chromosome Disorders, Congenital Abnormalities, +13 more
Locations
  • Neurocrine Clinical Site Phoenix, Arizona, United States
  • Neurocrine Clinical Site Los Angeles, California, United States
  • Neurocrine Clinical Site Sacramento, California, United States
  • Neurocrine Clinical Site Aurora, Colorado, United States
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Enrolling by Invitation

A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia

NCT05929807

Conditions
Achondroplasia, Bone Diseases, Bone Diseases, Developmental, +6 more
Locations
  • Ascendis Pharma Investigational Site Little Rock, Arkansas, United States
  • Ascendis Investigational Site Aurora, Colorado, United States
  • Ascendis Investigational Site Wilmington, Delaware, United States
  • Ascendis Investigational Site Saint Paul, Minnesota, United States
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Enrolling by Invitation

HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP

NCT05883748

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Erythropoietic Protoporphyria, +10 more
Locations
  • University of Alabama Hospital Birmingham, Alabama, United States
  • University of California San Francisco San Francisco, California, United States
  • University of Miami Miller School of Medicine Miami, Florida, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
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Enrolling by Invitation

A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)

NCT06908226

Conditions
Brain Diseases, Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +14 more
Locations
  • The Stanford Division of Child Neurology Palo Alto, California, United States
  • Childrens Hospital Colorado - PIN Aurora, Colorado, United States
  • University of Miami - 1120 NW 14th St Miami, Florida, United States
  • University of South Florida - 2 Tampa General Circle Tampa, Florida, United States
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Enrolling by Invitation

A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study

NCT05777993

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +10 more
Locations
  • McMaster Centre for Transfusion Medicine McMaster University Hamilton, Ontario, Canada
  • Tohoku University Hospital Sendai, Miyagi, Japan
  • Kyoto Katsura Hospital Kyoto, Japan
  • Toho University - Omori Medical Center Ōta-ku, Japan
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