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Clinical trial condition

Lysosomal Storage Diseases, Nervous System

Explore clinical trials studying Lysosomal Storage Diseases, Nervous System. Study availability and eligibility vary by location and protocol.

520 public trials 102 recruiting studies
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Lysosomal Storage Diseases, Nervous System trial results

Recruiting

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease

NCT06328608

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Phoenix Children's Phoenix, Arizona, United States
  • Emory Genetics Clinical Trials Center Atlanta, Georgia, United States
  • University of Iowa Iowa City, Iowa, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
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Recruiting

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

NCT07282847

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • Barrow Neurological Institute Phoenix, Arizona, United States
  • University of California, Irvine (UCI) Irvine, California, United States
  • Stanford Neuroscience Health Center Palo Alto, California, United States
  • NYU Langone New York, United States
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Recruiting

Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting

NCT07109375

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • AUSL Toscana Sud-Est - Ospedale San Donato Arezzo, Italy
  • Azienda Ospedaliera Universitaria Consorziale Policlinico Di Bari Bari, Italy
  • Irccs Azienda Ospedaliero-Universitaria Di Bologna - Policlinico Di Sant'Orsola Bologna, Italy
  • A.O. Sant'Anna e San Sebastiano Caserta, Italy
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Recruiting

Functional Capacity in Anderson-Fabry Disease Patients

NCT06884358

Conditions
Anderson-Fabry Disease, Brain Diseases, Brain Diseases, Metabolic, +20 more
Locations
  • Spedali Civili Hospital Brescia, Italy
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Milan, Italy
  • IRCCS Policlinico San Donato San Donato Milanese, Milan, Italy
  • Fondazione IRCCS San Gerardo dei Tintori Monza, Italy
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