Lipid Metabolism, Inborn Errors - 15 Studies Found

Completed : Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
:
  • Lipid Metabolism, Inborn Errors
  • Hypercholesterolemia, Autosomal
    : 2008-01-22
    :
    • Drug: mipomersen 200 mg mipome
Completed : Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia
:
  • Lipid Metabolism, Inborn Errors
  • Hypercholesterolemia, Autosomal
    : 2007-05-22
    : Drug: mipomersen sodium 200 mg/ml, in 1 ml solution for subcutaneous injection.
Enrolling by invitation : Natural History of Pearson Syndrome
: Pearson Syndrome
: 2014-12-22
Terminated : Phase 2 Study of EPI-743 in Children With Pearson Syndrome
: Pearson Syndrome
: 2014-04-02
: Drug: EPI-743 EPI-743 is the quinone oxidation product of alpha-tocotrienol
Completed : LYSO-PROVE - Determine the Prognostic Value of Lyso-Gb1 for Monitoring the Progress of Gaucher Disease
:
  • Lysosomal Storage Diseases
  • Gaucher Disease
  • Sphingolipidose
    : 2015-03-13
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