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 ...

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 ...

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 ...

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 ...