Rare Pediatric Disease Designation may render Pasithea eligible to receive a Priority Review Voucher (PRV)MIAMI, April 20, ...

Of the more than 3,000 variants that have been identified in the NF1 gene, only a handful of specific variants can be correlated to certain NF1 symptoms. In this post, I’ll discuss the major known ...

Neurofibromatosis 1 (NF1) is a rare genetic disorder that affects the nervous system, causing tumours to form on nerves throughout the body. It is also known as Von Recklinghausen disease, named after ...

A presentation at the 2026 ONA Summit described the role of oncology nurses in managing the care of patients with NF-1 and PN undergoing treatment with MEK inhibitors.

This genetic condition causes tumors to form on nerves throughout the body, often showing up as skin changes. The right treatment and management approach can help address symptoms and potential ...

Gomekli (mirdametinib) is the first treatment that is FDA approved for both adults and children with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas (PN). Selumetinib (Koselugo), a ...

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

Diagnostic criteria for neurofibromatosis type 1 (NF1) were established by a panel of experts at a National Institutes of Health (NIH) Consensus Development Conference in 1987. 1 A total of seven ...

Cred: SpringWorks Therapeutics. Mirdametinib is an inhibitor of mitogen-activated protein kinase kinases 1 and 2. Gomekli is the first medication approved for both adult and pediatric patients with ...

NF1 microdeletion syndrome is a common dominant genomic disorder responsible for around 5% of type I neurofibromatosis cases. The majority of cases are caused by mutations arising within the NF1 gene.