While this variability makes it difficult to know what to expect for a child with only café-au-lait spots, it’s important to understand the following points about complications related to NF1. During ...

Those medicines are not designed to eradicate or remove the plexiform neurofibromas or the neurofibromatosis, but they can slow down how it affects each individual patient. The earlier patients ...

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 following represents disclosure information provided by authors of this manuscript. All relationships are considered compensated unless otherwise noted. Relationships are self-held unless noted. I ...

The US Food and Drug Administration (FDA) has approved SpringWorks Therapeutics’ Gomekli (mirdametinib) to treat neurofibromatosis type 1 (NF1), a type of rare genetic disorder. Both adult and ...

“There are no treatments for many NF1 symptoms; current treatments for NF1 plexiform neurofibromas, such as surgery and MEK inhibitors, are helpful but come with their own set of trade ...

The single-arm trial focuses on oral investigational therapy’s efficacy and safety in NF1 subjects with plexiform neurofibromas (PNs), which are aggressive tumours that can lead to complications ...

SpringWorks Therapeutics portfolio features Ogsiveo for desmoid tumors and Gomekli for NF1‑plexiform neurofibromas. In fact, Ogsiveo’s revenues alone reached $172 million in 2024, supported by ...

for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.

Healx has dosed the first subject in the open-label Phase II INSPIRE-NF1 trial to assess the efficacy and safety of HLX-1502 ...