Mutations in NPC1 cause Niemann-Pick Type C. Mice lacking NPC1 only in microglia ... such as neuronal loss in the cerebellum and demyelination at late stages of the disease. Boland thinks these human ...

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 ...

“Dr. Berry-Kravis was able to make a request to the FDA so that Charlie could become the first human in the ... awareness of any rare disease,” Harry said. Niemann-Pick Disease is a rare ...

This discovery, published in Science, promises innovative therapeutic pathways to control cholesterol uptake, potentially reducing cardiovascular disease ... a protein, Niemann-Pick C1 Like ...

An FDA advisory committee has recommended approval of Zevra Therapeutics' arimoclomol for the ultra-rare disorder Niemann-Pick disease ... After the EMA's human medicines committee also said ...

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 WORLDSymposiumEight abstracts discussing data and clinical ...