Niemann-Pick Disease Type C Treatment

May 28, 2025

Niemann-Pick Disease (NPD) is a cluster of rare, genetically inherited lysosomal storage disorders characterized by the pathological accumulation of lipids within various organs. This buildup leads to progressive multisystem dysfunction, with severity and progression varying significantly across subtypes. While ind...

May 21, 2025

Lysosomal Storage Disorders, or LSDs, are rare genetic conditions that may not make headlines, but they affect thousands worldwide, often with life-changing consequences. LSDs happen when the body’s lysosomes, tiny cellular recycling centers, fail to break down waste materials due to missing or faulty enzymes. This...

May 02, 2025

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...

Nov 03, 2023

Niemann-Pick disease type C (NPC) is a rare genetic disorder caused by mutations in cholesterol trafficking proteins. There are two main types, with Type C1 being the most prevalent, accounting for around 95% of cases. While specific epidemiological data on NPC is limited, estimates suggest a prevalence of approxim...