For children with severe hypertrophic cardiomyopathy caused by gain-of-function RAS/mitogen-activated protein kinase (MAPK) mutations (RASopathy), trametinib reduces mortality and morbidity.
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 ...
Trametinib, a mitogen-activated protein kinase (MEK) inhibitor, reduces mortality and morbidity in children with severe hypertrophic cardiomyopathy (HCM) caused by pathogenic variants in the RAS/MAPK ...
Combined MEK and PI3K inhibition has had limited success in the clinic, although alternative methods of targeting these pathways may be effective. MEK inhibitors form the backbone of multiple ...
At this time, VSTM lives and dies on the fortunes of their inhibitor of MAPK signaling by deactivating the "downstream" regulators RAF and MEK, serving to choke off excess MAPK signaling (which ...