Tag: Mek inhibitor

Neurofibromatosis type 1 (NF1), a genetic disorder that causes tumors to grow on nerves, affects approximately 1 in every 3,000 people worldwide [1.6.3, 1.6.2]. For those affected, a critical question arises: what is the best medicine for neurofibroma?

In 2020, selumetinib received FDA approval as the first effective treatment for children with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PNs). But beyond its clinical success, a critical question remains: **how does selumetinib work** at a molecular level to produce these therapeutic effects?

Affecting approximately 1 in every 3,000 to 4,000 people, Neurofibromatosis type 1 (NF1) is a genetic disorder that can cause tumors to grow on nerves [1.5.7]. The newest answer to 'What is the new drug for NF1?' is mirdametinib (Gomekli), approved by the FDA in February 2025 [1.2.1].