Towards a Treatment for RAS-MAPK Pathway Disorders

The molecular pathogenesis of Noonan, Costello, and Cardio-facio-cutaneous syndromes has recently been described. All of these disorders result from an abnormal activation of the RAS-MAPK pathway. RAS-MAPK pathway activation is a common occurrence in tumor cells, and much effort has been made to develop inhibitors of this pathway to treat cancer. This chapter will describe several different strategies of RAS-MAPK pathway inhibition that are being evaluated in clinical trials. The potential application of these inhibitors to individuals with RAS-MAPK developmental disorders will also be discussed.

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