The Natural History of Type B Niemann-Pick Disease: Results From a 10-Year Longitudinal Study

Type B Niemann-Pick disease (NPD-B) caused by acid sphingomyelinase deficiency is a rare, autosomal recessive, lysosomal storage disorder with a broad range of disease severity. The objectives of this study were to document the natural history of the disease in a large, clinically heterogeneous pati...

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