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WORLDSymposium 2025 | Feb 3, 2025 - Feb 7, 2025
Acid Sphingomyelinase Deficiency (ASMD), historically known as Niemann-Pick disease (NPD) types A, A/B, and B, is a rare, autosomal recessive disease caused by pathogenic variants of the SMPD1 gene. In these patients, sphingomyelin accumulates in cells mainly of the mononuclear phagocytic system and the organs most affected include the liver, spleen, lungs, nervous system, and skeletal system.
