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Globally, NF1 affects approximately 1 in 3000 individuals. Plexiform neurofibromas (PN), a subtype of NF1-associated tumors, occur in up to 50% of patients with NF1.
The ReNeu trial for mirdametinib, a MEK inhibitor, demonstrated significant tumor shrinkage and quality-of-life improvements in patients with NF1-associated plexiform neurofibromas (NF1-PN).
Neurofibromatosis type 1 (NF1) is an autosomal-dominant genetic condition caused by loss-of-function variants in the NF1 gene, 1, 2 with a birth incidence of approximately 1 per 2,500. 3 NF1 variants ...
Overall, 53 of 114 patients met response criteria, defined as ≥20% reduction in target plexiform neurofibroma (PN) volume on consecutive MRI scans. Responses occurred in 24 of 58 adults and 29 ...
The randomised, global, multi-centre, double-blind, placebo-controlled trial enrolled 145 individuals with baseline characteristics, such as gender and distribution of PNs, representative of the ...
Dr. Brown added, “One of the biggest challenges in treating plexiform neurofibromas associated with NF1 is ensuring that patients remain on MEK inhibitor therapy over the long-term.
AstraZeneca (NASDAQ:AZN) and Merck (NYSE:MRK) reported positive topline results from a Phase 3 study of their drug Koselugo in the treatment of neurofibromatosis type 1, or NF1. The study showed ...
A rare genetic disorder known as Neurofibromatosis type one, or NF1, affects about one in 2,500 people in the U.S. That's about 120,000 people. Of those, half will develop a subtype--plexiform ...
Based on: The Treatment of Cavernous and Plexiform Angiomata by the Injection of Boiling Water (Wyeth Method). By Francis Reder, M.D., F.A.C.S. St. Louis: C. V. Mosby Company. 1918. Select the ...
The article by Moertel et al entitled “ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma” (J Clin ...