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Updated: May 16, 2026

Targeted Next-generation Sequencing and Bioinformatics Pipeline to Evaluate Genetic Determinants of Constitutional Disease
Published on: April 4, 2018
R542X mutation in SMPD1 gene: genetically novel mutation with phenotypic features intermediate between type A and
Aradhana Aneja1, Aditi Sharma, Ashwin Dalal
1Department of Pediatrics, Armed Forces Medical College, Pune, Maharashtra, India.
Abstract:
Niemann-Pick disease (NPD) is a heterogenous group of progressive neurovisceral disorder characterised by lysosomal accumulation of sphingomyelin. NPD types A and B are caused by mutations involving sphingomyelin-phosphodiesterase-1 (SMPD1) gene and are characterised by deficiency of acid sphingomyelinase activity. We present a case of a 9-month infant with clinical manifestations intermediate between types A and B NPD and genetically illustrating a novel R542X mutation in the exon 6 of SMPD1.
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