PUS3

Clinical Characteristics

The clinical features of this disease include:

  • Intellectual disability (severe to profound)
  • Global developmental delay
  • Failure to thrive
  • Growth deficiency (although birth growth parameters are usually normal)
  • Coarse facies
  • Microcephaly
  • Seizures (some patients)
  • Brain imaging abnormalities:
    • Ventriculomegaly
    • Arachnoid cysts
    • Brain atrophy
    • Dysgenesis of corpus callosum
    • T2/FLAIR signal abnormalities in the subcortical white matter
  • Grey sclera
  • Mongolian spots
  • Strabismus
  • Hypotonia