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Lysosomal Storage Diseases

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

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European Cystinosis Cohort 2

NCT07680751

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystinosis, Genetic Diseases, Inborn, +4 more
Locations: AP-HP_ Hôpital Charles Foix Ivry-sur-Seine, Île-de-France Region, France

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Morphology in Oral Rare Syndromes & Artificial Intelligence for Clinical Diagnosis

NCT07666269

Conditions: Artificial Intelligence (AI), Avitaminosis, Bone Diseases, +45 more
Locations: CHU de Bordeaux Bordeaux, France

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A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease

NCT07603050

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: Not specified

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Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF)

NCT07560956

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +23 more
Locations: Sahlgrenska University Hospital Gothenburg, Sweden

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Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)

NCT07530796

Conditions: Aspartylglucosamidase (AGA) Deficiency, Aspartylglucosamidase (AGA) deficiency, Aspartylglucosaminuria, +6 more
Locations: Not specified

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Study to Investigate the Efficacy of a Digestive Health Dietary Supplement on Post-meal Bloating in Healthy Participants

NCT07516587

Conditions: Abdominal Discomfort, Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +12 more
Locations: Alethios, Inc. San Francisco, California, United States

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Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial

NCT07479953

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations: Not specified

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A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion

NCT05669729

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: Not specified

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Care Pathway for Patients With Fabry's Disease (Fabry-PATH)

NCT07485660

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Service de Cardiologie - 1 Avenue du Professeur Jean Poulhès Toulouse, Occitanie, France

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Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis

NCT07445490

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations: Département de Neurologie Paris, France

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Lysosomal Storage Diseases

Lysosomal Storage Diseases trial results

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