GlobalData on MSN

FDA accepts Beren Therapeutics’ NDA for Niemann-Pick disease

The submission includes data indicating a meaningful survival benefit for infantile-onset NPC patients treated with adrabetadex.
Business Insider

Zevra Therapeutics Presents Positive New Real-World Data on MIPLYFFA® in Patients with Neimann-Pick Disease Type C (NPC) at the 22nd Annual WORLDSymposium™

CELEBRATION, Fla., Feb. 04, 2026 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with ...
Seeking Alpha

Zevra Therapeutics Celebrates and Supports Global Niemann-Pick Disease Awareness Day on October 19th and Niemann-Pick Disease Awareness Month Throughout October

CELEBRATION, Fla., Oct. 19, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a rare disease therapeutics company, proudly supports the Niemann-Pick community on Global Niemann-Pick ...
ALZFORUM

Multi-omics Spot Differences Between Pick’s and Alzheimer’s Diseases

In Pick’s disease, as in Alzheimer’s disease and frontotemporal dementia, tangled tau spells trouble. In this subtype of FTD, abnormal clumps of three-repeat tau called Pick bodies abound in neurons.
WLKY

Bowling fundraiser in Louisville supports 10-year-old’s fight against Niemann-Pick disease

DETAIL ALL THOSE THINGS IN YOUR SEVEN-DAY FORECAST. ERIC. THANK YOU. WELL, TO HELP THEIR DAUGHTER, ONE FAMILY IS MAKING IT THEIR MISSION TO FIND A CURE FOR NIEMANN-PICK DISEASE. IT’S A DEVASTATING ...