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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Ended earlyNCT03643562
What this trial is testing

Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

Who this might be right for
Niemann-Pick Type C Disease
Mandos LLC 27
Not applicableStudy completedNCT00414583
What this trial is testing

Stroke in Young Fabry Patients (sifap1): Frequency of Fabry Disease in Young Stroke Patients

Who this might be right for
Cerebrovascular Accident
CENTOGENE GmbH Rostock 5,000
Not applicableLooking for participantsNCT07235709
What this trial is testing

Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study

Who this might be right for
Fabry Disease
Yonsei University 25
Not applicableStudy completedNCT04577170
What this trial is testing

Pulsatility Index, Vasomotor Reactivity and Leukoencephalopathy in Fabry Patients

Who this might be right for
Fabry Disease
National and Kapodistrian University of Athens 45
Not applicableUnknownNCT05914727
What this trial is testing

Biomarkers Of Lung InVolvement In ASMD

Who this might be right for
Asmd, Visceral Type
Eline C. B. Eskes 68
Early research (Phase 1)Study completedNCT00151216
What this trial is testing

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

Who this might be right for
Batten DiseaseLate Infantile Neuronal Ceroid Lipofuscinosis
Weill Medical College of Cornell University 10
Not applicableUnknownNCT02852304
What this trial is testing

Assessment of the Prevalence of Lysosomal Acid Lipase Deficiency in Patients Waiting for a Liver Transplant.

Who this might be right for
Patients Waiting for a Liver Transplant.
Hospices Civils de Lyon 100
Testing effectiveness (Phase 2)Study completedNCT01907087
What this trial is testing

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

Who this might be right for
Jansky-Bielschowsky DiseaseBatten DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2+1 more
BioMarin Pharmaceutical 24
Testing effectiveness (Phase 2)Study completedNCT02068807
What this trial is testing

Evaluation of Antioxidant Activity of Oral Lutein in Preterm and Term Newborn

Who this might be right for
Infant, Newborn, DiseasesOther Lipid Storage Disorders
University of Siena 100
Not applicableStudy completedNCT00001972
What this trial is testing

PET Scan of Brain Metabolism in Relation to Age and Disease

Who this might be right for
Alzheimer's DiseaseBrain NeoplasmNiemann Pick Disease
National Institute of Neurological Disorders and Stroke (NINDS) 123
Testing effectiveness (Phase 2)Study completedNCT02402309
What this trial is testing

Topical NS2 Cream to Treat Ichthyosis in Sjögren-Larsson Syndrome (SLS)

Who this might be right for
Sjögren-Larsson Syndrome
Aldeyra Therapeutics, Inc. 12
Not applicableUnknownNCT02859363
What this trial is testing

Fabry Disease in Cerebrovascular Disease

Who this might be right for
Fabry Disease
Chang Gung Memorial Hospital 700
Early research (Phase 1)Looking for participantsNCT02254863
What this trial is testing

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Who this might be right for
AdrenoleukodystrophyBatten DiseaseMucopolysaccharidosis II+9 more
Joanne Kurtzberg, MD 40
Large-scale testing (Phase 3)Study completedNCT01842841
What this trial is testing

Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Shire 5
Not applicableUnknownNCT06065605
What this trial is testing

Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease

Who this might be right for
Fabry Disease
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 40
Testing effectiveness (Phase 2)Ended earlyNCT04145037
What this trial is testing

Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
AVROBIO 8
Not applicableUnknownNCT03590548
What this trial is testing

Clinical Audit of Managment of Gausher Disease in Children

Who this might be right for
Gaucher Disease
Yasmeen Abd Elbaset Hamzaa 1
Not applicableEnded earlyNCT06308718
What this trial is testing

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

Who this might be right for
Krabbe Disease
Forge Biologics, Inc 2
Testing effectiveness (Phase 2)Ended earlyNCT00004418
What this trial is testing

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Who this might be right for
Adrenoleukodystrophy
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 126
Early research (Phase 1)Ended earlyNCT00875160
What this trial is testing

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

Who this might be right for
Type 1 Gaucher Disease
Amicus Therapeutics 1
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