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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.

12,530 public trials 2,140 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Personalized Training for People With Rare Neuromuscular Disorders

NCT06708468

Conditions
Charcot Marie Tooth Disease (CMT), Charcot-Marie-Tooth Disease, Congenital Abnormalities, +20 more
Locations
  • Haukeland University Hospital Bergen, Norway
  • Helse Møre and Romsdal HF (Molde Hospital) Molde, Norway
  • Namsos Hospital Namsos, Norway
  • Oslo University Hospital Oslo, Norway
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Recruiting

Genotype/Phenotype Correlation of MORC2 Mutations

NCT07038239

Conditions
Charcot Marie Tooth Disease, Charcot-Marie-Tooth Disease, Congenital Abnormalities, +17 more
Locations
  • CHU de Besançon Besançon, France
  • CHRU Brest Brest, France
  • CHU Grenoble Grenoble, France
  • CH de Versailles Le Chesnay, France
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Recruiting

A Phase II Study of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa

NCT06628947

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Eye Diseases, Hereditary, +5 more
Locations
  • Save Sight Institute Sydney, New South Wales, Australia
  • Queensland Eye Institute Woolloongabba, Queensland, Australia
  • Royal Adelaide Hospital Adelaide, South Australia, Australia
  • Cerulea Clinical Trials East Melbourne, Victoria, Australia
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Recruiting

A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

NCT07399704

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +23 more
Locations
  • Associação Hospitalar de Prot à Infância Dr. Raul Carneiro Água Verde, Curitiba, Brazil
  • Hospital de Clinicas de Porto Alegre Porto Alegre, Rio Grande do Sul, Brazil
  • Instituto Nacional de Saúde da Mulher, da Criança e do Adolescente Fernandes Figueira Rio de Janeiro, Brazil
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Recruiting

Effectiveness and Safety of the Ulthera® System for Skin Laxity in the Lower Face, Submentum and Neck

NCT07614997

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, Cutis Laxa, +4 more
Locations
  • Merz Investigative Site Beijing, Beijing Municipality, China
  • Merz Investigative Site Beijing, Beijing Municipality, China
  • Merz Investigative Site Guangzhou, Guangdong, China
  • Merz Investigative Site Nanjing, Jiangsu, China
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Recruiting

Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

NCT07477691

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • UCLA Dept. of Human Genetics Los Angeles, California, United States
  • Children's Hospital Colorado Aurora, Colorado, United States
  • University Of South Florida Medical Center Tampa, Florida, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
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