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Clinical Trials for Metabolism, Inborn Errors

• Diesel Exhaust Induces Glucocorticoid Resistance
• Rifampin in CYP24A1-related Hypercalcemia and Hypercalciuria
• Effect of Allopurinol and Febuxostat on Urinary 2,8-Dihydroxyadenine Excretion
• Fatty Acid Oxidation Defects and Insulin Sensitivity
• Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases
• Randomised Controlled Trial of Mechanistic Effects of Rifaximin in Cirrhosis and Chronic Hepatic Encephalopathy
• Mendelian Reverse Cholesterol Transport Study
• Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
• Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate
• Pharmacogenetic Factors and Side Effects of Metoclopramide and Diphenhydramine
• Study to Investigate the Safety and Tolerability of AZD8848 in Butyrylcholinesterase Deficient Subjects
• Ataluren for Nonsense Mutation Methylmalonic Acidemia
• CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant
• Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects
• N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia
• Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
• An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia
• Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
• Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
• Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia
• Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration
• Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder
• Bezafibrate Trial in CPT2 Deficiency
• Nutritional Therapy of the Deficits of Oxidation Mitochondrial of the Fatty Acids
• Stem Cell Transplant for Inborn Errors of Metabolism
• Higher-Dose Ezetimibe to Treat Homozygous Sitosterolemia
• An Investigational Drug Study to Lower Non-Cholesterol Sterol Levels Associated With Sitosterolemia (0653-062)(COMPLETED)
• Investigational Drug in Patients With Hypercholesterolemia or in Patients With Sitosterolemia (0653-026)(COMPLETED)
• Sitosterolemia Extension Study (0653-004)(COMPLETED)
• Sitosterolemia Extension Study (0653-003)(COMPLETED)
• Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders
• Study of Phosphatidylcholine in a Patient With Methionine Adenosyltransferase Deficiency
• Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders
• Phase II Study of Ribose, Uridine, and Thymidine for a Complex Syndrome Involving Excessive 5'-Nucleotidase Activity
• Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome