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Niemann-Pick Disease Type C1 - 25 Studies Found
Estado | Estudiar |
Recruiting |
Nombre del estudio: Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C Condición: Neimann-Pick Disease, Type C Fecha: 2006-06-23 |
Recruiting |
Nombre del estudio: Biomarker for Niemann Pick Type C Disease Condición:
Fecha: 2011-03-01 |
Completed |
Nombre del estudio: Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients Condición: Human Acid Sphingomyelinase Deficiency Fecha: 2012-11-05 Intervenciones: Drug: Recombinant human acid sphingomyelinase Administered intravenously every 2 weeks for 26 weeks |
Completed |
Nombre del estudio: Miglustat in Niemann-Pick Type C Disease Condición: Niemann-Pick Type C Disease Fecha: 2007-08-15 Intervenciones: Drug: miglustat Patients aged 12 years or more randomised to the treatment group will receive 200 mg OGT |
Terminated |
Nombre del estudio: Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Condición:
Intervenciones: |
Recruiting |
Nombre del estudio: Genetic Characterization of Movement Disorders and Dementias Condición:
|
Completed |
Nombre del estudio: Application of Miglustat in Patients With Niemann-Pick Type C Condición: Niemann-Pick Disease Type C Fecha: 2013-01-02 Intervenciones: Drug: Miglustat miglustat 200mg tid for adult. For children surface area correction will be made. A lowe |