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

Selumetinib received FDA approval for adults with NF1 and symptomatic, inoperable plexiform neurofibromas, based on the phase 3 KOMET trial results. The KOMET trial showed a 20% objective response ...

GOMEKLI is the first FDA-approved treatment for adult and pediatric NF1-PN, showing effective tumor reduction and manageable safety. SpringWorks Therapeutics has announced the FDA approval of GOMEKLI ...

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 ...

Neurofibromatosis type 1 (NF1) is a hereditary condition arising from mutations in the NF1 gene, which encodes neurofibromin—a key regulator of cellular signalling and neural development. Children ...

RECKGIST score effectively predicts recurrence in neurofibromatosis type 1-associated GISTs, especially for tumors under 30 mm. Larger GISTs (over 30 mm) have a higher risk of metastasis, with ...

The FDA has approved selumetinib (Koselugo, AstraZeneca) for adults with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). Selumetinib, an oral kinase ...

Almost half of adults and children with neurofibromatosis type 1 (NF1) responded to the investigational MEK inhibitor mirdametinib, a large multicenter study of the rare condition showed. Overall, 53 ...

Despite what was previously thought, new research has shown that genetic changes alone cannot explain why and where tumors grow in those with genetic condition neurofibromatosis type 1 (NF-1).

There is currently no cure or treatment for NF1 that can reverse or prevent most complications. Because of this, medical management of the condition focuses on the early detection of treatable ...

A Pennsylvania mother and her one-year-old son share an unusual bond. They are both living with the same rare genetic disease. Lindsey Marson, 28, and her son, Bryson, were both born with ...

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