Zevra Therapeutics Executes Distribution Agreement to Broaden Access to MIPLYFFA® for the Treatment of Niemann-Pick Disease Type C (NPC)

NPC is an ultra-rare, relentlessly progressive, genetic disorder that leads to premature mortality. MIPLYFFA, used in conjunction with miglustat, is the only treatment shown to halt disease ...
Yahoo Finance

Niemann-Pick Disease Type C NPC Market Research 2025-2035 | Emerging Gene Therapies Offer New Hope As Zavesca and Trappsol Cyclo Manage Symptoms

Dublin, Aug. 01, 2025 (GLOBE NEWSWIRE) -- The "Niemann-Pick Disease Type C NPC Market - A Global and Regional Analysis: Focus on Country and Region - Analysis and Forecast, 2025-2035" report has been ...
The American Journal of Managed Care

Researchers Look at NPC Type C Prevalence in the United States

Niemann-Pick Disease Type C (NPC), an ultra-rare, progressive neurodegenerative disease, is heterogeneous and is often misdiagnosed or undiagnosed. A recent study aimed to get a fuller picture of the ...