Skip to main content
OpenTrials

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
Refine in advanced search Browse all conditions

Genetic Diseases, Inborn trial results

Enrolling by Invitation

Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy

NCT06066580

Conditions
Becker Muscular Dystrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +8 more
Locations
  • Arkansas Children's Hospital Little Rock, Arkansas, United States
  • UC San Diego La Jolla, California, United States
  • UCLA Medical Center Los Angeles, California, United States
  • UC Irvine Medical Center Orange, California, United States
View all 46 locations
View Trial Details
Enrolling by Invitation

A Study to Assess the Long-Term Safety and Efficacy of Lebrikizumab (LY3650150) in Participants 6 Months to <18 Years of Age With Moderate-to-Severe Atopic Dermatitis

NCT05735483

Conditions
Atopic Dermatitis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dermatitis, +10 more
Locations
  • Clinical Research Center of Alabama Birmingham, Alabama, United States
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Arkansas Research Trials North Little Rock, Arkansas, United States
  • First OC Dermatology Fountain Valley, California, United States
View all 94 locations
View Trial Details
Enrolling by Invitation

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT05368038

Conditions
Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +65 more
Locations
  • Maimonides Medical Center Brooklyn, New York, United States
  • NYU Langone Hospital - Brooklyn Brooklyn, New York, United States
  • North Shore University Hospital Manhasset, New York, United States
  • NYU Langone Health - Tisch Hospital New York, United States
View Trial Details
Enrolling by Invitation

Long-term Extension of GTX-102 in Angelman Syndrome

NCT06415344

Conditions
Abnormalities, Multiple, Angelman Syndrome, Central Nervous System Diseases, +7 more
Locations
  • University of California, Los Angeles (UCLA) Los Angeles, California, United States
  • University of California, San Diego - Rady Children's Hospital San Diego, California, United States
  • Rare Disease Research, LLC Atlanta, Georgia, United States
  • Rush University Medical Center Chicago, Illinois, United States
View all 23 locations
View Trial Details
Enrolling by Invitation

An Extension of SKY-0515 in Participants With Huntington's Disease

NCT07537075

Conditions
Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +14 more
Locations
  • John Hunter Hospital New Lambton Heights, New South Wales, Australia
  • Westmead Hospital Westmead, New South Wales, Australia
  • The University of Queensland Herston, Queensland, Australia
  • Flinders Medical Centre Adelaide, South Australia, Australia
View Trial Details
Enrolling by Invitation

An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy

NCT07573631

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne Muscular Dystrophy (DMD), Genetic Diseases, Inborn, +8 more
Locations
  • Fondazione Serena ETS - Centro Clinico NeMO Milano Milan, Italy
  • UOC Fase I - Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore Rome, Italy
  • Leids Universitair Medisch Centrum Leiden, Netherlands
  • Hospital Viamed Santa Angela De la Cruz Seville, Spain
View Trial Details

Related conditions