Niemann-Pick disease type A

Niemann-Pick disease type A is a very severe subtype of Niemann-Pick disease, an autosomal recessive lysosomal disease, and is characterized clinically by onset in infancy or early childhood with failure to thrive, hepatosplenomegaly, and rapidly progressive neurodegenerative disorders.

Also known as: Niemann-PICK disease, type A, Niemann-Pick disease, Intermediate, protracted neurovisceral, sphingomyelin lipidosis, sphingomyelinase deficiency

61 clinical trials for this condition and its sub-types.