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 […]

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.

The Gilbert Family Foundation (GFF) recently established a partnership with Sage Bionetworks (Sage) to enable the storage, management, and sharing of data generated by GFF-funded research.

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 […]

The Gilbert Family Foundation is excited to announce the launch of its second research institutive to accelerate the development of therapies for neurofibromatosis type 1 (NF1). Around 1 in 5 NF1 patients develop optic pathway gliomas (OPGs) that cause degradation of the optic nerve and subsequent vision loss. […]

The Gilbert Family Foundation's seeks to eradicate neurofibromatosis type 1 (NF1). With our first initiative, we plan to accelerate the development of curative therapies that address the underlying genetic abnormalities in NF1 patients.