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.

Shares of SpringWorks Therapeutics SWTX have rallied 32.9% in the past three months compared with the industry’s increase of ...

The start of human dosing in a clinical trial Monday, plus a drug approval earlier this month, together offer the potential to reshape the treatment of the rare disease neurofibromatosis type 1 (NF1).

Despite its impact, treatment options remain limited, with many patients facing invasive surgeries or therapies with severe side effects. A Potential Breakthrough for NF1 Patients “This trial ...

Genetic changes alone don’t fully explain tumor growth in NF-1, pointing to other contributing factors. This insight may ...

RECKGIST score may predict recurrence in neurofibromatosis type 1 GISTs, potentially guiding treatment decisions, according ...

Discover a study that used the Skindex-29 assessment to evaluate emotional wellbeing during selumetinib cycles.

and treatment of cancers." More information: T. R. W. Oliver, et al. Cancer-independent, second somatic NF1 mutation of normal tissues in neurofibromatosis type 1, Nature Genetics (2025).

Researchers have found in a nonrandomised pilot trial that selumetinib showed a reduction in cutaneous neurofibromatosis ...