Preclinical models provide a key role in developing treatments for neurofibromatosis type 1. The Gilbert Family Foundation not only supports projects that utilize available preclinical models, but projects that aim to expand these resources.

The Gilbert Family Foundation today announced funding of a new $5.4 million clinical study which aims to understand the mechanisms of vision loss caused by NF1-associated optic pathway gliomas, or NF1-OPGs.

The Gilbert Family Foundation today announced the launch of a clinical study that aims to validate optical coherence tomography (OCT) as a tool to objectively assess the visual system and its response to treatment in NF1 patients with optic pathway gliomas.

The Gilbert Family Foundation (GFF) is excited to announce the development of a database for neurofibromatosis type 1 (NF1) research tools in collaboration with Sage Bionetworks. The database aims to support the development of a robust NF1 research toolkit while also lowering the barrier of entry to research for new-to-NF1 […]

GFF's Gene Therapy Initiative seeks to explore and develop gene-targeting therapeutic strategies for neurofibromatosis type 1 (NF1) that address the underlying cause of the disease. Targeting the mutated gene itself could yield a more effective, comprehensive response for a greater range of patients.

Optic pathway gliomas (OPGs) are slow-growing brain tumors that arise in or around the optic nerve and can cause the degeneration of the optic nerve and retinal ganglion cells (RGCs) leading to vision loss. GFF's Vision Restoration Initiative aims to develop new therapies to protect and restore vision for […]

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 […]

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. […]