Joubert syndrome: monozygotic twins with discordant phenotypes

J Child Neurol. 1999 Oct;14(10):649-54; discussion 669-72 discussio. doi: 10.1177/088307389901401005.

Abstract

We describe three sisters with Joubert syndrome, two of whom are monozygotic twins with highly discordant phenotypes. The twins were born at 34 weeks' gestation with discordant birthweights. Their anatomic, neurologic, and developmental status differs greatly: Twin B is able to walk, run, and is verbal, unlike Twin A who is wheelchair-bound, severely retarded, nonverbal, and autistic. Abnormal eye movements and retinal dysplasia are striking features in all three girls, but none has renal cysts seen by ultrasonography. Magnetic resonance images show the "molar tooth sign," the radiologic hallmark of Joubert syndrome, although only one twin, the most severely handicapped, has severe hypoplasia of the cerebellar hemispheres. Phenotypic differences between the twins could be attributable to postzygotic unequal division of the inner cell mass, unequal sharing of the venous return from a monochorionic placenta, mosaicism, or a mutation of a modifying gene.

MeSH terms

  • Cerebellum / abnormalities*
  • Child
  • Child, Preschool
  • Developmental Disabilities / diagnosis
  • Developmental Disabilities / genetics*
  • Diseases in Twins / genetics*
  • Female
  • Fetal Growth Retardation / diagnosis
  • Fetal Growth Retardation / genetics
  • Follow-Up Studies
  • Fourth Ventricle / abnormalities
  • Fourth Ventricle / pathology
  • Genetic Predisposition to Disease / genetics*
  • Humans
  • Infant
  • Magnetic Resonance Imaging
  • Neurologic Examination
  • Phenotype*
  • Pregnancy
  • Spinocerebellar Ataxias / diagnosis
  • Spinocerebellar Ataxias / genetics*
  • Syndrome
  • Twins, Monozygotic