Category: Genetic Disorders / Lysosomal Storage Disorder
Niemann-Pick disease is a group of rare, inherited metabolic disorders characterized by the accumulation of lipids (fats) within cells, leading to progressive organ damage and neurological dysfunction.
Type A/B: 1 in 250,000 births; Type C: 1 in 100,000 to 1 in 150,000 births (Source: NORD)
Discover the latest clinical trials and treatments for Niemann-Pick Disease on JourneyForHealth.
View Full Analysis