Neurofibromatosis Type 1 (NF1)
NF1, which stands for Neurofibromatosis type 1 and has historically been called von Recklinghausen's disease, is a group of genetic disorders that cause tumors to form on nerve tissue. These tumors can develop anywhere in the nervous system, including the brain, spinal cord and nerves.
Symptoms vary widely between patients, but can include multiple brown spots on the skin (café-au-lait macules), freckling in the underarm or groin, plexiform neurofibromas (non-cancerous, slow growing tumors involving the nerve sheaths), and yellowish-brown lesions on the colored portion of the eye.
The diagnosis of NF1 is usually made during the first decade of life, based on characteristic skin freckling, cafe-au-lait spots, optic glioma and/or pseudoarthrosis.
Koselugo (selumetinib)
Koselugo (selumetinib)
Koselugo (selumetinib) is a MEK inhibitor indicated for the treatment of children 2 years of age and older with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas that cannot be completely removed by surgery.
Patient Resources
National Organization for Rare Disorders (NORD) supports patient advocacy, education, and research.
The Neurofibromatosis Network is a non-profit organization dedicated to finding treatments and a cure for neurofibromatosis by promoting scientific research, improving clinical care, providing outreach through education and awareness, while offering hope and support to those affected by NF.
The Children’s Tumor Foundation (CTF) Ending Neurofibromatosis Through Research is a not-for-profit, voluntary organization dedicated to improving the well-being of individuals and families affected by neurofibromatosis type I (NF1) and type II (NF2).
OneSource (Personalized Patient Support from Alexion) provides links to patient advocacy groups and access to a patient education manager to help patients learn more about their disease through local and community education sessions and events.
