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 ...
Friday, the FDA approved Zevra Therapeutics, Inc.'s (NASDAQ:ZVRA) Miplyffa (arimoclomol), an oral medication for Neimann-Pick disease, type C (NPC). Miplyffa, in combination with Johnson & Johnson's ...
As the Sept. 21 PDUFA date looms for arimoclomol from Zevra Therapeutics Inc. in Niemann-Pick type C (NPC), the U.S. FDA’s newly formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) decided ...
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 ...
Coherus and Chinese partner Junshi Biosciences announced U.S. FDA approval of Loqtorzi in all lines of treatment for nasopharyngeal carcinoma (NPC). Coherus management has insisted they will "not ...
LAKELAND, Fla. (Ivanhoe Newswire) --- 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 ...
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