Nasdaq: FDA Approves SpringWorks Therapeutics' GOMEKLI for Treatment of Neurofibromatosis Type 1 in Adults and Children FDA Approves SpringWorks Therapeutics' GOMEKLI for Treatment of Neurofibromatosis Type 1 in Adults and Children Learning that your child has neurofibromatosis type 1 (NF1) comes with a range of emotions and practical concerns. While many people diagnosed with NF1 will have a normal life expectancy, the ... EurekAlert!: An international study validates the efficacy of a drug in adults with neurofibromatosis type 1 An international study validates the efficacy of a drug in adults with neurofibromatosis type 1 The US Food and Drug Administration (FDA) has approved mirdametinib (Gomekli, SpringWorks Therapeutics, Inc.) for certain adult and pediatric patients with neurofibromatosis type 1 (NF1).
Specifically ... Neurofibromatosis type 1 (NF1) is a genetically inherited disorder that manifests in a broad spectrum of clinical features including dermatological, neurological, skeletal and notably, ocular ... 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 ...
