Disease Score gda Association Type Type Original DB Sentence supporting the association PMID PMID Year
CUI: C0220756
Disease: Niemann-Pick Disease, Type C
Niemann-Pick Disease, Type C
0.010 Biomarker disease BEFREE Miglustat, which reversibly inhibits glucosylceramide synthase, reportedly has beneficial effects on the progressive neurological symptoms of NPC and was approved in Japan in 2012. 28587793 2017