CHICAGO -- Selumetinib (Koselugo) significantly improved response rates in adults with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas, the phase III KOMET trial ...

As a new year begins, plans are underway for the 8 th Annual Rare Disease Genomics Symposium to be held on February 26 th and 27 th. The two-day event will be virtual this year to limit the spread of ...

These are rare tumors that grow from nerve cells. They form on the protective cover around your nerves. That’s called the sheath. Most plexiform neurofibromas (PNs) are benign, or non-cancerous. Some ...

This month, our discussion focuses on the clinical management of plexiform neurofibromas in the era of selumetinib, a MEK inhibitor medication that was approved by the FDA in 2020 for the treatment of ...

A new preclinical model of pulmonary arterial hypertension (PAH) may lead to improved research and ultimately better therapies for this life-threatening problem, according to its developers, ...

Christopher L. Moertel, MD, University of Minnesota, discusses the trial data that helped get mirdametinib approved and what providers can do to keep measuring quality of life (QOL) improvements. The ...

Plexiform neurofibromas are tumors that grow along nerves. That’s how they get their name: "neuro" means nerves, and "fibroma" is a type of tumor. These growths have nerve tissue and many different ...

Twenty-four of 58 adults (41%) and 29 of 56 children (52%) had a BICR-confirmed objective response during the 24-cycle treatment phase; in addition, two adults and one child had confirmed responses ...

Effective medical therapies are lacking for the treatment of neurofibromatosis type 1–related plexiform neurofibromas, which are characterized by elevated RAS–mitogen-activated protein kinase (MAPK) ...

A new preclinical model of pulmonary arterial hypertension (PAH) may lead to improved research and ultimately better therapies for this life-threatening problem, according to its developers. A new ...