164 studies found for:    Open Studies | "Brain Diseases, Metabolic, Inborn"
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Open Studies | "Brain Diseases, Metabolic, Inborn"
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Rank Status Study
1 Recruiting Biomarker for Metachromatic Leukodystrophy Disease
Conditions: Leukodystrophy, Metachromatic;   Hereditary Central Nervous System Demyelinating Diseases;   Brain Diseases, Metabolic, Inborn;   Sphingolipidoses;   Lysosomal Storage Diseases, Nervous System
Intervention:
2 Recruiting Longitudinal Study of Urea Cycle Disorders
Conditions: Brain Diseases, Metabolic, Inborn;   Amino Acid Metabolism, Inborn Errors;   Urea Cycle Disorders
Intervention:
3 Recruiting UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Conditions: Adrenoleukodystrophy;   Batten Disease;   Mucopolysaccharidosis II;   Leukodystrophy, Globoid Cell;   Leukodystrophy, Metachromatic;   Neimann Pick Disease;   Pelizaeus-Merzbacher Disease;   Sandhoff Disease;   Tay-Sachs Disease;   Brain Diseases, Metabolic, Inborn
Intervention: Biological: DUOC-01
4 Recruiting Natural History Study of Children With Metachromatic Leukodystrophy
Conditions: Leukodystrophy, Metachromatic;   Hereditary Central Nervous System Demyelinating Diseases;   Brain Diseases, Metabolic, Inborn;   Brain Diseases, Metabolic;   Brain Diseases;   Central Nervous System Diseases;   Nervous System Diseases;   Sulfatidosis;   Sphingolipidoses;   Lysosomal Storage Diseases, Nervous System;   Leukoencephalopathies;   Demyelinating Diseases;   Metabolism, Inborn Errors;   Genetic Diseases, Inborn;   Lipidoses;   Lipid Metabolism, Inborn Errors;   Lysosomal Storage Diseases;   Metabolic Diseases;   Lipid Metabolism Disorders
Intervention: Other: Natural History Study of Children With Metachromatic Leukodystrophy
5 Recruiting Biomarker for Krabbe Disease
Conditions: Lysosomal Storage Diseases;   Krabbe Disease
Intervention:
6 Recruiting Protein Sorbent Properties of Montmorillonite in Vitro and in Vivo Models
Conditions: Urea Cycle Disorders, Inborn;   Other Metabolic Diseases
Interventions: Dietary Supplement: montmorillonite 5 g;   Dietary Supplement: montmorillonite 3 g;   Dietary Supplement: Montmorillonite 1 g
7 Unknown  Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease
Condition: Fabry Disease
Intervention: Drug: recombinant alpha-galactosidase A
8 Recruiting The Impact of Artichoke Leaf Extract on Blood Cholesterol: Primary Study
Condition: Lipid Metabolism Disorders
Interventions: Dietary Supplement: Artichoke Leaf Extract;   Other: Placebo
9 Recruiting Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
Condition: Pompe Disease
Interventions: Other: Thoracic MRI;   Other: Spirometry;   Other: Inspiratory Load Compensation;   Other: Maximal Inspiratory Pressure;   Other: Resting Breathing Pattern;   Other: Respiratory Muscle Endurance Test
10 Not yet recruiting Betaine METABOLISM OF PATIENTS With Homocystinuria
Condition: Homocystinuria
Intervention: Drug: Betaine
11 Recruiting Home Therapy With VPRIV in Gaucher's Disease
Condition: Gaucher's Disease
Intervention:
12 Recruiting Antioxidant Signature in Adult Patients With Phenylketonuria
Conditions: PKU;   Hyperphenylalaninemia
Interventions: Other: Blood sample;   Other: Urine sample
13 Recruiting Exploration of Immunity in Gaucher Disease
Condition: Gaucher Disease
Intervention:
14 Recruiting Examining the Immune Response in Patients With Gaucher Disease and Hepatitis C
Conditions: Gaucher Disease;   Hepatitis C
Intervention:
15 Recruiting Androgenetic Alopecia in Fabry Disease
Condition: Fabry Disease
Intervention:
16 Recruiting Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
Condition: Pompe Disease
Intervention:
17 Recruiting Investigating Lysosomal Storage Diseases in Minority Groups
Conditions: Lysosomal Storage Disorders;   Gaucher Disease;   Fabry Disease;   Pompe Disease;   Niemann-Pick Disease
Intervention:
18 Recruiting Genes Involved in Lipid Disorders
Condition: Dyslipidemia
Intervention:
19 Recruiting Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
Condition: Niemann-Pick Disease, Type C1
Intervention: Drug: 2-hydroxypropyl-beta-cyclodextrin
20 Recruiting Fabry and Cardiomyopathy (FaCard) Epidemiological Study for the Analysis of Biomarkers and the Clinical Course of Patients With Fabry Disease and the N215S-mutation
Conditions: Cerebrovascular Accident;   Stroke, Acute;   Cerebral Stroke
Intervention:

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Indicates status has not been verified in more than two years