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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Not applicableLooking for participantsNCT05843552
What this trial is testing

Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease

Who this might be right for
Gaucher Disease
University of Minnesota 30
Not applicableUnknownNCT02000310
What this trial is testing

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

Who this might be right for
Lysosomal Storage Disorders
O & O Alpan LLC 80
Early research (Phase 1)Ended earlyNCT02231710
What this trial is testing

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

Who this might be right for
Primary Immune Deficiency DisordersHemophagocytic LymphohistiocytosisInherited Bone Marrow Failure Syndrome+2 more
Bellicum Pharmaceuticals 1
Testing effectiveness (Phase 2)Not Yet RecruitingNCT06819514
What this trial is testing

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

Who this might be right for
Fabry Disease
Guangzhou Jiayin Biotech Ltd 16
Not applicableStudy completedNCT01271699
What this trial is testing

Prevalence of Fabry Disease in a Defined Population at Risk - Patients Formerly Diagnosed With Multiple Sclerosis

Who this might be right for
Multiple Sclerosis
CENTOGENE GmbH Rostock 250
Not applicableLooking for participantsNCT03362164
What this trial is testing

Evaluation of HEArt invoLvement in Patients With FABRY Disease

Who this might be right for
Rare DiseasesFabry DiseaseFabry Disease, Cardiac Variant+1 more
Wuerzburg University Hospital 650
Not applicableLooking for participantsNCT06607471
What this trial is testing

Multimodal and Multidisciplinary Approach to Optimize Diagnostic, Prognostic, and Therapeutic Management of Patients with Non-ischemic Cardiomyopathies and Arrhythmogenic-inflammatory Phenotypes: a Multicenter, Observational, Retrospective and Prospective Registry Study.

Who this might be right for
Non-ischemic CardiomyopathyDilated Cardiomyopathy (DCM)Hypertrophic Cardiomyopathy (HCM)+12 more
Scientific Institute San Raffaele 15,000
Not applicableLooking for participantsNCT00344331
What this trial is testing

Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

Who this might be right for
Niemann-Pick Disease, Type C
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 900
Not applicableStudy completedNCT04281537
What this trial is testing

Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

Who this might be right for
Fabry Disease
Amicus Therapeutics 82
Not applicableUnknownNCT02843334
What this trial is testing

Study of the Prevalence of Fabry Disease in French Dialysis Patients

Who this might be right for
Fabry DiseaseEnd Stage Renal DiseaseRenal Dialysis
Hospices Civils de Lyon 6,000
Not applicableStudy completedNCT01947634
What this trial is testing

Sleepiness and Sleep-disordered Breathing in Fabry Disease. A Prospective Cohort Study.

Who this might be right for
Fabry Disease
University of Zurich 52
Not applicableLooking for participantsNCT05588167
What this trial is testing

Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

Who this might be right for
Niemann-Pick Disease, Type C
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 100
Not applicableStudy completedNCT02650219
What this trial is testing

Auto-antibodies Prevalence and CD1 Role in Gaucher Disease

Who this might be right for
Gaucher Disease
Hospital St. Joseph, Marseille, France 60
Testing effectiveness (Phase 2)UnknownNCT02559830
What this trial is testing

Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy

Who this might be right for
Metachromatic LeukodystrophyAdrenoleukodystrophy
Shenzhen Second People's Hospital 50
Not applicableNot Yet RecruitingNCT07445490
What this trial is testing

Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis

Who this might be right for
Tay-Sachs Disease GangliosideSandhoff Disease Ganglioside
Assistance Publique - Hôpitaux de Paris 6
Not applicableStudy completedNCT01182818
What this trial is testing

Fabry and Stroke Epidemiological Protocol (FASEP): Risk Factors In Ischemic Stroke Patients With Fabry Disease

Who this might be right for
Cerebrovascular AccidentStroke, AcuteCerebral Stroke
University of Rostock 1,000
Post-approval studies (Phase 4)Study completedNCT00097890
What this trial is testing

Replagal Enzyme Replacement Therapy for Adults With Fabry Disease

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 25
Not applicableStudy completedNCT01417520
What this trial is testing

Clinical and Pathophysiological Investigations Into Erdheim Chester Disease

Who this might be right for
MyelofibrosisGaucher DiseasePulmonary Fibrosis+2 more
National Human Genome Research Institute (NHGRI) 88
Testing effectiveness (Phase 2)Active Not RecruitingNCT05152914
What this trial is testing

Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

Who this might be right for
Neuronal Ceroid Lipofuscinosis Type 2
David L Rogers, MD 5
Large-scale testing (Phase 3)Looking for participantsNCT05710692
What this trial is testing

Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease

Who this might be right for
Fabry Disease
Chiesi Farmaceutici S.p.A. 16
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