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Clinical Trials for Lipid Metabolism, Inborn Errors

• Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
• Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
• Fatty Acid Oxidation Defects and Insulin Sensitivity
• Phase 2 Study of EPI-743 in Children With Pearson Syndrome
• Effects of Fish Oil and Colesevelam (STAIR7007)
• Mendelian Reverse Cholesterol Transport Study
• Sitosterolemia Metabolism
• The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM)
• Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
• Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects
• 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
• 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
• 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)