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