We conducted an open-label, phase 2 trial of selumetinib to determine the objective response rate among patients with plexiform neurofibromas and to assess clinical benefit. Children with ...
A Central Valley single mother is facing an uphill battle to save her 4-year-old daughter from a rare disease that could ...
This month, our discussion focuses on the clinical management of plexiform neurofibromas in the era of selumetinib, a MEK inhibitor medication that was approved by the FDA in 2020 for the treatment of ...
A 45-year-old lady, originally from Surinam, presented with a 3 year history of epiphora and irritation of both eyes. There was no other relevant past medical or family history. Visual acuity was ...
NEUROFIBROMATOSIS is a congenital and hereditary disorder involving ectodermal, neuroectodermal and mesodermal tissue. Café-au-lait spots, cutaneous neurofibromas, tumors of the central and peripheral ...
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 ...