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 ...

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 ...

Findings from the ReNeu trial showed treatment with mirdametinib resulted in statistically significant objective response rates. The Food and Drug Administration (FDA) has accepted for Priority Review ...

Neurofibromatosis type 1 (NF1) is a prevalent genetic disorder caused by germline mutations in the NF1 gene, leading to dysregulation of RAS signalling and predisposing affected individuals to diverse ...

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

SpringWorks Therapeutics, a Merck company, announced that the European Commission has granted conditional marketing authorization for EZMEKLY® (mirdametinib) for the treatment of symptomatic, ...

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 ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Koselugo’s approval was based on data from the phase 3 ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Mirdametinib is an investigational mitogen-activated ...

Mirdametinib received FDA approval for NF1 patients aged 2 and older, showing efficacy in reducing PN volume and improving quality of life. Conditional European approval was granted for mirdametinib, ...

While the first medical treatment for neurofibromatosis (NF) was recently approved, many further options are on the horizon. "You see a massive acceleration of research and development," said Annette ...

PHILADELPHIA (WPVI) -- There's no cure for neurofibromatosis (NF), and it's been said that the rare genetic disorder is becoming more common. Now a local mother, who was born with the rare disease, is ...