Niemann-Pick disease

Search Trials
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients
Status:
Active, not recruiting
Last Changed:
May 1, 2019
First Changed:
Nov 17, 2014
Disease(s):
Sphingomyelin Lipidosis
Intervention(s):
Olipudase alfa
Induced Pluripotent Stem Cells for Niemann Pick Disease
Status:
Recruiting
Last Changed:
Apr 25, 2019
First Changed:
Mar 21, 2019
Disease(s):
Niemann-Pick Diseases
Biomarker for Niemann Pick Type C Disease (BioNPC)
Status:
Recruiting
Last Changed:
May 14, 2019
First Changed:
Mar 2, 2011
Disease(s):
Niemann-Pick Disease
Open‐Label Study of Long‐Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann‐Pick Disease Type C
Status:
Recruiting
Last Changed:
Mar 28, 2019
First Changed:
Mar 28, 2019
Disease(s):
Niemann-Pick Disease, Type C1
Intervention(s):
Hydroxypropyl-β-cyclodextrin
Safety and Efficacy of Miglustat in Chinese NPC Patients
Status:
Not yet recruiting
Last Changed:
Sep 4, 2019
First Changed:
Apr 10, 2019
Disease(s):
Niemann-Pick Disease, Type C
Intervention(s):
Miglustat

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