Curing NF

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Watch: Vision Restoration Initiative Annual Meeting Brings “Dream Team” Together to Advance Therapeutic Research

Recently, our Vision Restoration Initiative (VRI) awardees and their teams convened for our VRI Annual Meeting to discuss the results of their research projects focused on vision restoration therapies. The teams presented their data for Phase A, the first set of discovery projects that concluded earlier this year, as well […]
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Gilbert Family Foundation Furthers Commitment to Curing Neurofibromatosis (NF), Invests Additional $18 Million Toward Gene Therapy Initiative

The Gilbert Family Foundation is excited to announce a new round of three-year grants in the Gene Therapy Initiative to support the development of curative therapies that address the underlying cause of NF1.
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Leveraging Preclinical Models to Develop Therapies for NF1

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.
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Gilbert Family Foundation Funds Clinical Study to Understand Vision Loss from NF1 Optic Pathway Glioma

The Gilbert Family Foundation is excited to announce a $5.4 million study to understand the mechanisms of vision loss caused by NF1-associated optic pathway gliomas, or NF1-OPGs. Led by Drs. Robert Avery (CHOP) and Jeffrey Goldberg (Stanford), findings are expected to inform the development of future therapies, validate existing […]
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Gilbert Family Foundation Collaborates with the Children’s Oncology Group and Children’s Hospital of Philadelphia to Validate a New Tool to Measure Progressive Vision Loss

The Gilbert Family Foundation (GFF) 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.
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GFF and Sage Bionetworks Collaborate on an NF1 Research Tools Database

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 […]
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Seeking a True Cure: Developing Gene Replacement and Editing Therapies to Address the Cause of 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.
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GFF’s Vision Restoration Initiative: Exploring Regenerative Therapies

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 […]
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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 […]
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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 […]
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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.
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