MPDZ

Clinical Characteristics

The clinical features of this disease, per organ system, include:

Central Nervous System (CNS)

  • Nonobstructive hydrocephalus
  • Variably dilated ventricles
  • Thinning of the corpus callosum
  • Thinning of the cerebral cortex
  • Lissencephaly
  • Colpocephaly
  • Simplified gyral patterns
  • Gray matter heterotopia
  • Intellectual disability (reported in some patients)
  • Seizures (reported in some patients)
  • Gray matter heterotopia (reported in some patients)

Head and Neck

  • Macrocephaly
  • Variable dysmorphic facial features (reported in some patients), including:
    • Asymmetric face
    • Frontal bossing
    • Downslanting palpebral fissures
    • Posteriorly rotated ears
    • Bulbous nose
    • Microretrognathia
  • Iris coloboma
  • Strabismus
  • Macular hypoplasia
  • Foveal dysplasia
  • Optic atrophy (reported in some patients)
  • Sensorineural hearing loss (reported in one patient)

Cardiovascular

  • Cardiac septal defects
  • Aberrant subclavian artery (reported in one patient)
  • Portal vein thrombosis (reported in one patient)

Other(s) (rare)

  • 2-vessel cord
  • Polyhydramnios
  • Congenital diaphragmatic hernia
  • Lung hypoplasia
  • Malrotation of the gut
  • Multicystic dysplastic kidney

The disease has a highly variable severity, where some patients may die in infancy while others may have normal neurological development.