Lipid Metabolism, Inborn Errors - 25 Studies Found
Recruiting |
: LYSO-PROVE - Determine the Prognostic Value of Lyso-Gb1 for Monitoring the Progress of Gaucher Disease :
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Recruiting |
: International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related Diseases : Neutral Lipid Storage Disease : 2016-08-04 |
Completed |
: The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM) : Neutral Lipid Storage Disease : 2012-01-16 : Drug: Fibrate treatment Patients will receive a dosage of 400mg Bezafibrate every day during 28 weeks |
Recruiting |
: Clinical Study on the Safety of CNT-02 for TGCV and NLSD-M :
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Active, not recruiting |
: Safety and Efficacy Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency : Lysosomal Acid Lipase Deficiency : 2014-03-20 : Drug: sebelipase alfa Intravenous infusion of sebelipase alfa: 1mg/kg every other week |