TBCK

Management

The management for this rare disorder at this time is limited to supportive care, as there is no known cure to date.

  • All patients with TBCK-related encephalopathy should be evaluated for developmental delays and started on appropriate therapies. Physical, occupational and speech therapies are recommended to maximize their developmental potential.
  • Seizures should be treated symptomatically, with no specific anti-epileptic medication currently recommended.
  • Feeding support should be provided, if children have difficulties swallowing.
  • Particularly in children with the more severe presentation of the disease, there is evidence that other symptoms, in addition to neurological, can be present, and children should be screened for thyroid function (hypothyroidism has been found), elevated lipids (high cholesterol or triglycerides) and bone health (osteopenia and osteoporosis also reported).
  • There should also be a careful evaluation for visual impairment and hearing loss.

Targeted therapies are currently in preclinical trials.