Infantile spinal muscular atrophy with respiratory distress type 1: a case report

J Child Neurol. 2010 Jun;25(6):764-9. doi: 10.1177/0883073809344121. Epub 2010 Mar 1.

Abstract

The condition, currently known as spinal muscular atrophy with respiratory distress type 1, is an unusual variant of spinal muscular atrophy type 1 that is characterized by early respiratory failure due to diaphragmatic paralysis. The defective gene, the immunoglobulin mu-binding protein 2 (IGHMBP2 gene), of this autosomal recessive disorder is located on chromosome 11q13 and encodes immunoglobulin mu-binding protein 2. The natural history and phenotypic spectrum of the disease are still not clear. The authors present the first genetically proven case of spinal muscular atrophy with respiratory distress type 1 to be reported from Saudi Arabia. The parents are first cousins and the causative gene sequencing revealed mutation in exon 7 reported for the first time in a homozygous form. The clinical scenario of the case is discussed. The findings in the muscle magnetic resonance imaging (MRI) are presented.

Publication types

  • Case Reports

MeSH terms

  • DNA-Binding Proteins / genetics*
  • Fatal Outcome
  • Female
  • Homozygote
  • Humans
  • Infant
  • Magnetic Resonance Imaging
  • Muscle, Skeletal / pathology
  • Pedigree
  • Saudi Arabia
  • Spinal Muscular Atrophies of Childhood / genetics*
  • Spinal Muscular Atrophies of Childhood / pathology
  • Transcription Factors / genetics*

Substances

  • DNA-Binding Proteins
  • IGHMBP2 protein, human
  • Transcription Factors