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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.

12,537 public trials 2,139 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia

NCT00912041

Conditions
Amyotrophic Lateral Sclerosis, Brain Diseases, Brain Infarction, +38 more
Locations
  • University of California, Davis Sacramento, California, United States
  • Stanford University School of Medicine Stanford, California, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
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Recruiting

A Study to Investigate APL-4098 Alone and in Combination in Adults With AML or MDS

NCT06372717

Conditions
Acute Myeloid Leukemia (AML), Acute Myeloid Leukemia Refractory, Acute Myeloid Leukemia, in Relapse, +17 more
Locations
  • Monash Health Clayton, Victoria, Australia
  • St. Vincent's Hospital Melbourne Fitzroy, Victoria, Australia
  • The Alfred Hospital Melbourne, Victoria, Australia
  • Hollywood Private Hospital Nedlands, Western Australia, Australia
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Recruiting

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

NCT06844214

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +9 more
Locations
  • University of Florida, 2004 Mowry Road - Site Number: 8400005 Gainesville, Florida, United States
  • University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001 Tampa, Florida, United States
  • Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003 New York, United States
  • Virginia Commonwealth University Medical Center- Site Number : 8400006 Richmond, Virginia, United States
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Recruiting

Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

NCT05040373

Conditions
Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +15 more
Locations
  • Clinical Trial Site Iowa City, Iowa, United States
  • Clinical Trial Site Nantes, France
  • Clinical Trial Site Münster, Germany
  • Clinical Trial Site Pavia, Italy
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Recruiting

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

NCT03128996

Conditions
Anemia, Anemia, Hemolytic, Anemia, Hemolytic, Congenital, +16 more
Locations
  • Yale School of Medicine New Haven, Connecticut, United States
  • Nemours Children's Health Wilmington, Delaware, United States
  • Helen DeVos Children's Hospital Grand Rapids, Michigan, United States
  • Washington University School of Medicine St Louis, Missouri, United States
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Recruiting

Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1

NCT06188741

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +17 more
Locations
  • Childrens of Alabama Birmingham, Alabama, United States
  • Children's Hospital of Los Angeles Los Angeles, California, United States
  • Stanford University Palo Alto, California, United States
  • Children's National Hospital Washington D.C., District of Columbia, United States
View all 15 locations
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Recruiting

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

NCT04561518

Conditions
ATTR Amyloidosis, Amyloid Neuropathies, Amyloid Neuropathies, Familial, +16 more
Locations
  • Clinical Trial Site La Jolla, California, United States
  • Clinical Trial Site Los Angeles, California, United States
  • Clinical Trial Site Jacksonville, Florida, United States
  • Clinical Trial Site Iowa City, Iowa, United States
View all 40 locations
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Recruiting

Donidalorsen Treatment in Children With Hereditary Angioedema

NCT07298447

Conditions
Angioedema, Angioedemas, Hereditary, Cardiovascular Diseases, +14 more
Locations
  • Ionis Investigative Site Santa Monica, California, United States
  • Ionis Investigative Site Miami, Florida, United States
  • Ionis Investigative Site St Louis, Missouri, United States
  • Ionis Investigative Site Cincinnati, Ohio, United States
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