For children with severe hypertrophic cardiomyopathy caused by gain-of-function RAS/mitogen-activated protein kinase (MAPK) mutations (RASopathy), trametinib reduces mortality and morbidity.
1Hereditary Cancer Program, Catalan Institute of Oncology (ICO-IDIBELL), Hospitalet de Llobregat, Barcelona, Spain. 2Program in Molecular Mechanisms and Experimental Therapy in Oncology (Oncobell), ...
Affected children are born with café-au-lait spots and sometimes Lisch nodules. During adolescence and young adulthood, more than 19% of affected individuals with NF1 develop cutaneous neurofibromas ...
This is the fourth FDA-approved indication for Braftovi, coming after it was cleared in combination with MEK Inhibitor Mektovi (binimetinib) for BRAF-mutated melanoma and non-small cell lung ...
an oral RAF/MEK clamp, in combination with defactinib, an oral FAK inhibitor, for the treatment of adult patients with recurrent low-grade serous ovarian cancer (LGSOC), w Shoshana Shendelman ...