Infants less than six months old with Noonan Syndrome, hypertrophic cardiomyopathy and congestive heart failure normally have a poor prognosis, with a one-year survival rate of 34 percent. In the new study, doctors used Trametinib to try to treat NS in two patients. They observed dramatic improvement of clinical and cardiac status in the patients only three months after treatment.
A promising new treatment for infants with Noonan syndrome
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