Niemann-Pick disease treatment: a systematic review of clinical trials

Niemann-Pick (NP) disease is caused by an abnormality in lysosomes, which are unable to degrade macromolecules; as a result, the latter accumulate inside these organelles to form cellular inclusions (1-3). NP disease encompasses a set of autosomal recessive hereditary abnormalities (1) characterized by the accumulation of lipids, mostly sphingomyelin and cholesterol, in different organs such as spleen or liver (1,4,5)