PHGDH

Clinical Characteristics

Mutations in PHGDH gene may cause the following signs and symptoms:

Head

  • Microcephaly (abnormally small head)

Neck

  • Short neck

Face

  • Sloping forehead
  • Undersized jaw
  • Protruding eyes
  • Increased distance between the eyes
  • Clouding of the lens of the eye
  • Absent eyelids
  • Abnormally small eyes
  • Absent eyelashes
  • Large ears
  • Flattened nose
  • Thick lips
  • Gaping mouth
  • Cleft palate and cleft lip

Cardiovascular

  • Defects of the walls of the heart
  • Other congenital anomalies of the heart

Respiratory

  • Lung underdevelopment

Skeletal

  • Short limbs
  • Joint contractures
  • Pterygia (pinkish, triangular tissue growth on the cornea of the eye)
  • Toes and/or fingers that failed to separate and/or were bent (camptodactyly)
  • Wedge-shaped fifth finger
  • Puffiness of hands and feet
  • Poorly mineralized bones

Skin

  • Thin, scaly yellow skin
  • Absent eyelashes
  • Absent scalp hair

Central Nervous System

  • Absence of brain folds
  • Underdevelopment or absence of corpus callosum (the part that connects the two hemispheres of the brain together)
  • Underdevelopment or absence of the cerebellum (the part of the brain mainly responsible for coordinating movement)
  • Absence of nerves responsible for sense of smell
  • Spina bifida (incomplete closing of the backbone and the membranes around the spinal cord
  • Dandy-walker malformation (a congenital anomaly affecting mainly the cerebellum and causing fluid accumulation and increased pressure inside the skull, which may to multiple brain function problems)

Other

  • Excess of amniotic fluid in the amniotic sac
  • Short umbilical cord
  • Small placenta