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Maroc Medical. 2012; 34 (4): 267-270
in French | IMEMR | ID: emr-151572

ABSTRACT

Niemann Pick disease type B is a rare autosomal recessive lipid storage disorder, characterized by a partial deficiency of sphingomyelinase We report two cases one in a boy aged of 3 years old, and the other a 18-month-old girl. All of them have related parents. The circumstances of discovery were a hepato-splenomegaly without neurological signs and interstitial syndrome on chest radiograph. Laboratory tests showed a moderate hepatic cytolysis and microcytic hypochromic anemia. The bone marrow showed many cells overload Niemann-Pick type. Diagnostic confirmation was achieved by a decrease in cell lysosomal enzyme activity. It is a lysosomal disease characterized by visceral abnomality with preserved nervous system function. The current treatment of Niemann Pick disease is purely symptomatic awaiting the use of enzymatic replacement therapy

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