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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.

12,584 public trials 2,158 recruiting studies
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Genetic Diseases, Inborn trial results

Active, not recruiting

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

NCT05335876

Conditions
Atrophy, Bulbo-Spinal Atrophy, X-Linked, Central Nervous System Diseases, +19 more
Locations
  • Child Hosp Of The Kings Daughters Norfolk, Virginia, United States
  • Novartis Investigative Site Sydney, New South Wales, Australia
  • Novartis Investigative Site Leuven, Belgium
  • Novartis Investigative Site Curitiba, Paraná, Brazil
View all 32 locations
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Active, not recruiting

Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

NCT06627179

Conditions
Abnormalities, Multiple, Blindness, Congenital Abnormalities, +28 more
Locations
  • The University of California, San Francisco San Francisco, California, United States
  • Bascom Palmer Eye Institute/University of Miami Miami, Florida, United States
  • Emory University Atlanta, Georgia, United States
  • Massachusetts Eye and Ear Boston, Massachusetts, United States
View all 26 locations
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Active, not recruiting

An International, Multicenter, Randomized, Double-Blind, Parallel Group, Vehicle-Controlled, Phase 2/3 Study With Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1% Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS)

NCT06073132

Conditions
Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Epidermolysis Bullosa, +8 more
Locations
  • Mission dermatology Center Rancho Santa Margarita, California, United States
  • Stanford University Stanford, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • Northwestern University - Lurie Childrens's Hospital Chicago, Illinois, United States
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Active, not recruiting

A Trial to Investigate Teprotumumab Subcutaneous Administration Compared With Placebo in Male and Female Adult Participants With Moderate-to-severe Active Thyroid Eye Disease

NCT06248619

Conditions
Autoimmune Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Endocrine System Diseases, +12 more
Locations
  • Catalina Eye Care - NVISION - PPDS Tucson, Arizona, United States
  • Advanced Quality Medical Research Orland Park, Illinois, United States
  • W Kellogg Eye Center Ann Arbor, Michigan, United States
  • Las Vegas Endocrinology Henderson, Nevada, United States
View all 38 locations
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