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Niemann-Pick disease
A group of inherited, severe metabolic disorders in which sphingomyelin accumulates in lysosomes in cells. The lysosomes normally transport material through and out of the cell.
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Niemann-Pick disease type C 12 trials · 13 incl. sub-types Sub-types →
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Acid sphingomyelinase deficiency 7 trials · 10 incl. sub-types Sub-types →
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Niemann-Pick disease type E 0 trials