GFF’s Vision Restoration Initiative: Exploring Neuroprotection/Neuroenhancement Therapies
Approximately 1 in 5 patients with neurofibromatosis type 1 (NF1) develop optic pathway gliomas (OPGs). These tumors can cause degeneration of the optic nerve and retinal ganglion cells (RGCs), ultimately leading to vision loss. GFF's Vision Restoration Initiative aims to develop neuroprotection/neuroenhancement therapies that protect and increase performance of injured RGCs and the optic nerve to improve visual function.
GFF’s Guiding Values: Collaboration
The Gilbert Family Foundation (GFF) is on a mission to eradicate neurofibromatosis type 1 (NF1), but we can't do it alone. The GFF Curing NF research programs have been designed with collaboration at the core to align with other NF1 focused organizations and ensure all researchers have opportunities to share and advance together.
Nothing Nonsensical About Nonsense Mutations: GTI Researchers Target One of the Most Common Types of NF1 Disease-Causing Genetic Mutations
While more than 1000 different mutations of the neurofibromatosis type 1 (NF1) gene have been identified to cause the disease, 1 in 5 are classified as "nonsense mutations". Several projects in GFF's Gene Therapy Initiative are exploring and identifying potential therapeutics to address NF1 nonsense mutations.
FDA Approves Selumetinib – What it Means for the NF Community and What’s Next?
On April 10, 2020, the FDA approved the first drug that was ever developed for neurofibromatosis type 1 (NF1). Selumetinib (aka AstraZeneca’s Koselugo) has been approved to treat symptomatic and inoperative NF1-associated plexiform neurofibromas and is currently in the midst of clinical trials to evaluate the possible benefits to other NF1-associated tumors.