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Chanarin-Dorfman Syndrome - 7 Studies Found
Estado | Estudiar |
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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Recruiting |
Nombre del estudio: Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy Condición:
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Active, not recruiting |
Nombre del estudio: Clinical Trial in Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency Condición: Lysosomal Acid Lipase Deficiency Fecha: 2014-07-07 Intervenciones: Drug: sebelipase alfa Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL). The investig |
Terminated |
Nombre del estudio: Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102 Condición:
Fecha: 2011-11-10 Intervenciones: Drug: SBC-102 SBC-102 is a recombinant human lysosomal acid lipase (rhLAL). The investigational medicina |
Active, not recruiting |
Nombre del estudio: SurVival of Lysosomal Acid Lipase Deficiency (LAL-D) Infants Treated With SebelipAse aLfa Condición:
Fecha: 2011-06-09 Intervenciones: Drug: Sebelipase alfa (SBC-102) Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL). Th |