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Clinical trial condition

Heredodegenerative Disorders, Nervous System

Explore clinical trials studying Heredodegenerative Disorders, Nervous System. Study availability and eligibility vary by location and protocol.

1,876 public trials 411 recruiting studies
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Heredodegenerative Disorders, Nervous System trial results

Active, Not Recruiting

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

NCT04251026

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +22 more
Locations
  • UCSF Benioff Children's Hospital Oakland, California, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • UNC Children's Research Institute Chapel Hill, North Carolina, United States
  • UPMC | Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
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Active, Not Recruiting

An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

NCT04348136

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +16 more
Locations
  • Fukui Clinical site Fukui, Japan
  • Fukuoka Clinical site 2 Fukuoka, Japan
  • Fukuoka Clinical site Fukuoka, Japan
  • Gifu Clinical site Gifu, Japan
View all 20 locations
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Active, Not Recruiting

An Observational Study Using Artificial Intelligence (AI) Algorithms on Electrocardiography (ECG), Point-of-care Ultrasound (POCUS), and Transthoracic Echocardiophy (TTE) to Estimate the Under-diagnosis of Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Across a Diverse Range of US Health Systems.

NCT07062848

Conditions
Amyloid Neuropathies, Amyloid Neuropathies, Familial, Amyloidosis, +13 more
Locations
  • University of California - San Francisco (UCSF) Health San Francisco, California, United States
  • Yale New Haven Health System New Haven, Connecticut, United States
  • Henry Ford Health Detroit, Michigan, United States
  • Mount Sinai New York, United States
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Active, Not Recruiting

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

NCT05901987

Conditions
Central Nervous System Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +9 more
Locations
  • Peking University， First Hospital, Department of Pediatrics Beijing, China
  • Bayi Children's Hospital, Seventh Medical Center, PLA general hospital Beijing, China
  • West China Second University Hospital, Sichuan University Chengdu, China
  • Children's Hospital of Chongqing Medical University Chongqing, China
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