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 ...

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 ...

Zevra to receive a 2025 New Treatment Award for MIPLYFFA™ at the 21st Annual WORLDSymposium Eight abstracts discussing data and clinical experience associated with MIPLYFFA accepted for poster ...

Privately-held IntraBio's Aqneursa also won FDA approval for the treatment of Niemann-Pick disease type C ... for people with advanced small-cell lung cancer. It is the first and only so ...