INDIANAPOLIS (WISH) — Niemann-Pick Type C, or NPC, is a relentlessly progressive and fatal genetic disorder that impacts both the body and the brain. In the U.S., only about 900 people are living with ...

A Zevra Therapeutics drug is now the first FDA-approved therapy for an ultra-rare, inherited metabolic disorder whose effects on the central nervous system can become fatal by the time a patient is a ...

MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative disease MIPLYFFA is indicated for use in combination with miglustat ...

CENTOGENE and Insilico Medicine will work together to identify, rank, and annotate proteins to identify novel therapeutic targets for NPC using AI NPC is a rare genetic disease occurring in ...