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