AQNEURSA
May 02, 2025
New Era in Niemann-Pick Disease: A Comparative Review of MIPLYFFA and AQNEURSA
Niemann-Pick disease type C (NPC) is a rare, aggressive, and life-threatening neurovisceral disorder with a profound impact on both patients and the healthcare system. Caused by mutations in the NPC1 or NPC2 genes, NPC disrupts intracellular lipid transport, leading to toxic accumulation of cholesterol and other li...
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