Niemann-Pick disease is a rare genetic medical condition. There are four variants of this disease, categorized as type A, type B, type C, and type D. Niemann-Pick disease causes a variety of ...

Niemann-Pick disease affects multiple systems throughout the body. It has many symptoms, and the severity varies from case to case. Rather than being one disease, Niemann-Pick has four distinct types.

Mutations in NPC1 cause Niemann-Pick Type C. Mice lacking NPC1 only in microglia have rampant neuroinflammation ... such as neuronal loss in the cerebellum and demyelination at late stages of 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 Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease. The US regulator has cleared Miplyffa (arimoclomol ...

Exosomes, nano-sized extracellular vesicles secreted by all cells, are a fundamental part of the human body’s internal ... development for Niemann–Pick disease type C, a disorder in which ...

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 ...