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Clinical trial condition

Lysosomal Storage Diseases

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

710 public trials 130 recruiting studies
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Lysosomal Storage Diseases trial results

Completed

Intracerebral Gene Therapy for Sanfilippo Type A Syndrome

NCT01474343

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +13 more
Locations
  • Hôpital Bicêtre - Assistance Publique des Hôpitaux de Paris Le Kremlin-Bicêtre, France
  • Hôpital Necker, Assistance Publique des Hôpitaux de Paris Paris, France
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Completed

A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients

NCT00144768

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +12 more
Locations
  • Childrens Hospital Los Angeles Los Angles, California, United States
  • Connecticut Children's Medical Center Hartford, Connecticut, United States
  • Children's Hospital of Wisconsin Milwaukee, Wisconsin, United States
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Completed

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

NCT01387854

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations
  • Children's Hospital and Research Center Oakland, California, United States
  • Women's and Children's hospital North Adelaide, Australia
  • Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
  • Hôpital Femme Mère Enfant Lyon, France
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Completed

High Dose or High Dose Frequency Study of Alglucosidase Alfa

NCT00483379

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Birmingham, Alabama, United States
  • Stanford, California, United States
  • Washington D.C., District of Columbia, United States
  • Chicago, Illinois, United States
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