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Sphingolipidoses

Clinical trial condition

Sphingolipidoses

Explore clinical trials studying Sphingolipidoses. Study availability and eligibility vary by location and protocol.

394 public trials 77 recruiting studies

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Sphingolipidoses trial results

Not yet recruiting

National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children

NCT06985212

Conditions: Acid Sphingomyelinase Deficiency (ASMD), Brain Diseases, Brain Diseases, Metabolic, +20 more
Locations: Groupe Hospitalier Diaconesses Croix Saint-Simon Paris, France

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Not yet recruiting

Study of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease

NCT06869499

Conditions: Blood Platelet Disorders, Brain Diseases, Brain Diseases, Metabolic, +33 more
Locations: Groupe Hospitalier Diaconesses Croix Saint-Simon Paris, France

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Not yet recruiting

Aortic Dimensions in Patients With Fabry Disease

NCT06956573

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Not specified

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Not yet recruiting

The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease

NCT06819514

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: General Hospital of Eastern Theater Command Nanjing, Jiangsu, China

Children's Hospital Zhejiang University School of Medicine Hangzhou, Zhejiang, China

First Affiliated Hospital of Zhejiang University Hangzhou, Zhejiang, China

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Not yet recruiting

Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3

NCT06523517

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: Peking union medical college hospital Beijing, China

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Not yet recruiting

Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency

NCT06258577

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: China Medical University Hospita Taichung, Taiwan

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Enrolling by invitation

Genetic Studies of Lysosomal Storage Disorders

NCT00001215

Conditions: Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +21 more
Locations: National Institutes of Health Clinical Center Bethesda, Maryland, United States

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Enrolling by invitation

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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Enrolling by invitation

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT05368038

Conditions: Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +65 more
Locations: Maimonides Medical Center Brooklyn, New York, United States

NYU Langone Hospital - Brooklyn Brooklyn, New York, United States

North Shore University Hospital Manhasset, New York, United States

NYU Langone Health - Tisch Hospital New York, United States

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Enrolling by invitation

Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease

NCT06880250

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: #13 Astrakhan, Russia

#27 Barnaul, Russia

#7 Borovichi, Russia

#6 Chelyabinsk, Russia

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Sphingolipidoses

Sphingolipidoses trial results

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