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Found 25 studies with search of: "Hereditary Central Nervous System Demyelinating Diseases" OR "megalencephalic leukoencephalopathy with subcortical cysts"
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Rank Status Study
1 Active, not recruiting Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Conditions: I Cell Disease;   Fucosidosis;   Globoid Cell Leukodystrophy;   Adrenoleukodystrophy;   Mannosidosis;   Niemann-Pick Disease;   Pulmonary Complications;   Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Metachromatic Leukodystrophy;   Gaucher's Disease;   Wolman Disease
Intervention:
2 Recruiting Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
Condition: Canavan Disease
Interventions: Dietary Supplement: GTA (Glyceryl triacetate);   Drug: GTA glyceryl triacetate
3 Completed Stem Cell Transplant for Inborn Errors of Metabolism
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Gaucher's Disease;   Fucosidosis;   Wolman Disease;   Niemann-Pick Disease;   Batten Disease;   GM1 Gangliosidosis;   Tay Sachs Disease;   Sandhoff Disease
Interventions: Procedure: Stem Cell Transplant;   Drug: Busulfan, Cyclophosphamide, ATG
4 Active, not recruiting GTA-Glyceryltriacetate for Canavan Disease
Conditions: Infantile Canavan Disease;   Deficiency Disease, Aspartoacylase
Intervention: Drug: GTA: Glyceryltriacetate
5 Enrolling by invitation Compassionate Use of Metazym in Patients With Late Infantile Metachromatic Leukodystrophy
Condition: Late Infantile Metachromatic Leukodystrophy
Intervention: Drug: rhASA
6 Completed Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dehydroepiandrosterone Replacement for Primary Adrenal Insufficiency
Condition: Addison's Disease
Intervention: Drug: dehydroepiandrosterone
7 Recruiting Study of Glyceryl Trierucate and Glyceryl Trioleate (Lorenzo's Oil) Therapy in Male Children With Adrenoleukodystrophy
Condition: Adrenoleukodystrophy
Interventions: Drug: glyceryl trierucate;   Drug: glyceryl trioleate
8 Completed Survey in Patients With Chronic Adrenal Insufficiency in Germany
Conditions: Adrenal Insufficiency;   Addison's Disease
Intervention:
9 Completed Study of Oral Cholic Acid in Patients With Inborn Errors of Bile Acid Synthesis
Conditions: Infantile Refsum's Disease;   Zellweger Syndrome;   Hyperpipecolic Acidemia;   Adrenoleukodystrophy;   Peroxisomal Disorders;   Cholestasis;   Bifunctional Enzyme Deficiency
Intervention:
10 Recruiting The Natural History of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention:
11 Recruiting Adrenal Function After Living Kidney Donation
Conditions: Addison Disease;   Hypoaldosteronism
Intervention: Procedure: Laparoscopic hand-assisted donor nephrectomy
12 Completed Long-Term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Condition: Late Infantile Metachromatic Leukodystrophy
Intervention: Drug: rhASA
13 Not yet recruiting Study of Bile Acids in Patients With Peroxisomal Disorders
Conditions: Infantile Refsum's Disease;   Zellweger Syndrome;   Bifunctional Enzyme Deficiency;   Adrenoleukodystrophy
Interventions: Drug: chenodeoxycholic acid;   Drug: cholic acid;   Drug: ursodiol
14 Recruiting Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children
Condition: Adrenoleukodystrophy
Intervention: Procedure: blood sampling and medical records
15 Recruiting HSCT for High Risk Inherited Inborn Errors
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Tay Sachs Disease;   Sandhoffs Disease;   I-Cell Disease;   Sanfillipo;   GM1 Gangliosidosis
Interventions: Drug: Campath;   Drug: Clofarabine;   Procedure: Total body Irradiation;   Drug: Melphalan
16 Suspended Lithium and Acetate for Canavan Disease
Conditions: Canavan Disease;   Infantile;   Deficiency Disease;   Aspartoacylase;   Leukodystrophy, Spongiform
Intervention: Drug: Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug)
17 Completed Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
Condition: Adrenoleukodystrophy
Interventions: Drug: glyceryl trierucate;   Drug: glyceryl trioleate;   Drug: interferon beta;   Drug: thalidomide
18 Completed Diagnostic and Screening Study of Genetic Disorders
Conditions: Tay-Sachs Disease;   Porphyria, Erythropoietic;   Leukodystrophy, Globoid Cell;   Metabolism, Inborn Errors
Intervention:
19 Completed Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Condition: Metachromatic Leukodystrophy
Intervention: Drug: rhASA
20 Recruiting Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
Conditions: Hurler's Syndrome;   Maroteaux-Lamy Syndrome;   Sly Syndrome;   Alpha Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Sphingolipidoses;   Krabbe Disease;   Wolman's Disease;   Niemann-Pick Disease Type B;   Niemann-Pick Disease, Type C
Interventions: Procedure: Stem Cell Transplantation;   Drug: Campath, Busulfan, Cyclophosphamide

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