which causes sphingomyelin to build up in the cells, which in turn interferes with the function of several organs in the body. All of the types of Niemann-Pick disease are autosomal recessive ...

Mutations in NPC1 cause Niemann-Pick Type C. Mice lacking NPC1 only in microglia have rampant neuroinflammation ... found that inactivating Npc1 only in myeloid cells could recapitulate many aspects ...

In the space of four days, the number of FDA-approved therapies for ultra-rare genetic disorder Niemann-Pick disease has gone from zero to two, transforming the prospects for patients. IntraBio's ...

Zevra Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease ... other lipids within cells. That leads to the ...

Exosomes, nano-sized extracellular vesicles secreted by all cells, are a fundamental ... product is in preclinical development for Niemann–Pick disease type C, a disorder in which a defective ...