Because NF1 is an unpredictable disorder, it varies widely in severity – even among two people in the same family. Some people with NF1 may be completely unaware that they are affected, experiencing ...

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, a drug that ...

It can spawn tumors throughout the nervous system and bone deformities that riddle the whole body with pain. One of the most common rare diseases in the world, the genetic condition neurofibromatosis ...

Neurofibromatosis type 1 (NF1) is a genetic condition that causes tumors to grow along nerves in the skin, brain, and other parts of the body. These tumors are usually noncancerous (benign), but they ...

Most therapeutic approaches for NF1 have focused on blocking a specific cell signaling pathway, called Ras/MAPK, which is hyperactive in cells that have lost NF1 function due to gene mutation. This ...

Panelists discuss how neurofibromatosis type 1 (NF1) is typically diagnosed in early childhood using National Institutes of Health (NIH) clinical criteria, with genetic testing supporting unclear ...

Treatment for neurofibromatosis type 1 may involve surgery, radiation, chemotherapy, or other methods. In some cases, doctors may take a “wait and see” approach. Neurofibromatosis type 1 (NF1), ...

Spinal deformities such as scoliosis and kyphosis are among the most serious complications of neurofibromatosis type 1 (NF1), a genetic disorder that affects about one in 3,000 people. These ...

The US Food and Drug Administration has approved mirdametinib for the treatment of neurofibromatosis type 1 (NF1) in adults and children. The drug, developed by SpringWorks Therapeutics, is the first ...

Most NF1-PNs cannot be fully removed with surgery or other treatments. Therefore, it’s important to take steps to manage the condition and reach out to others for support as you enter adulthood. Many ...