In June 2022, the FDA granted Accelerated Approval to the BRAF inhibitor dabrafenib in
combination with the MEK inhibitor trametinib for the treatment of adult and paediatric …

In the era of precision medicine, it has been increasingly recognized that individuals with a
certain disease are complex and different from each other. Due to the underestimation of the …

Activating RAS mutations are found in a subset of fusion-negative rhabdomyosarcoma
(RMS), and therapeutic strategies to directly target RAS in these tumors have been …

Purpose: Malignant peripheral nerve sheath tumors (MPNST) are lethal, Ras-driven
sarcomas that lack effective therapies. We investigated effects of targeting cyclin-dependent …

Highlights•PNs are a major cause of morbidity in NF1.•Promising efficacy results with MEK
inhibitors and cabozantinib have been reported.•MEK inhibitors are associated with a …

Introduction NF1 is a tumor suppressor gene encoding neurofibromin, an inhibitor of the
RAS/MAPK and PI3K-AKT-mTOR signaling pathways. NF1 germline pathogenic variants …

Abstract MAP/ERK kinase 1 and 2 (MEK 1/2) inhibitors (MEKi) are investigated in several
trials to treat lesions that arise from pathogenic variants of the Neurofibromatosis type 1 and …

Congenital pseudarthrosis of the tibia (CPT) is a severe pathology marked by spontaneous
bone fractures that fail to heal, leading to fibrous nonunion. Half of patients with CPT are …

Despite the promising antitumor activity of RAF/MEK inhibitors for RAS-driven cancers, not
all patients respond to these therapies. Adaptive resistance has been reported as a major …

Malignant peripheral nerve sheath tumors (MPNSTs) are highly aggressive soft tissue
sarcomas with limited treatment options, and new effective therapeutic strategies are …