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Successful treatment of refractory chylothorax with MEK inhibitor trametinib in a child with Noonan syndrome: case report.

Ines HribernikTeresa BrooksAlix Dunlop-JonesJames R Bentham
Published in: European heart journal. Case reports (2023)
MEK inhibition with trametinib is emerging as a possible salvage treatment option for a subset of patients with Noonan syndrome and severe pulmonary lymphangiectasia. More experience is required to establish optimal treatment regimen and long-term outcomes.
Keyphrases
  • case report
  • mental health
  • pulmonary hypertension
  • early onset
  • cell proliferation
  • replacement therapy