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 ...

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 ...

Comprehensive submission package includes evidence of substantially improved survival among adrabetadex-treated patients with infantile-onset Niemann Pick disease type C (NPC), intended to serve as a ...

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 ...