Early-onset neurodegenerative disease of the cerebellum and motor axons

Pediatr Neurol. 2009 May;40(5):365-70. doi: 10.1016/j.pediatrneurol.2008.11.020.

Abstract

We describe a novel hereditary neurodegenerative disease of infancy affecting an Aboriginal family from northern Manitoba, Canada. The parents are nonconsanguineous, without a family history of neurodegenerative diseases. Four of 10 siblings (three males and one female) presented with neurologic abnormalities including arthrogryposis, seizures, and severe developmental delay shortly after birth. In two children, cerebellar atrophy and mild cerebral atrophy were documented on neuroimaging. Two children, a boy who died at age 40 months and a girl who died at age 22 months, underwent muscle biopsies at 3 weeks and 4 months of age, respectively. The biopsies revealed fiber-size variability in the boy, and grouped atrophy with fiber-type grouping in the girl. Two boys who died at ages 7.5 and 37 months underwent autopsies that indicated severe atrophy of the cerebellar hemispheres (especially the inferior lobules and vermis), hypomyelination of white-matter fascicles in the striatum, severe atrophy of corticospinal tracts in the brainstem and spinal cord, and atrophy of the anterior spinal roots. In the spinal cord, motor neuron cell bodies and the posterior columns were spared. This clinical entity likely represents a novel neurodegenerative disease of the cerebellum and long motor axons.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Atrophy
  • Brain / pathology
  • Cerebellar Diseases / complications*
  • Cerebellar Diseases / mortality
  • Cerebellar Diseases / pathology*
  • Child, Preschool
  • Family
  • Female
  • Heredodegenerative Disorders, Nervous System / complications*
  • Heredodegenerative Disorders, Nervous System / mortality
  • Heredodegenerative Disorders, Nervous System / pathology*
  • Humans
  • Indians, North American
  • Infant
  • Infant, Newborn
  • Magnetic Resonance Imaging
  • Male
  • Motor Neuron Disease / complications*
  • Motor Neuron Disease / mortality
  • Motor Neuron Disease / pathology*
  • Motor Neurons / pathology
  • Muscles / pathology
  • Pedigree
  • Phenotype
  • Photomicrography
  • Spinal Cord / pathology