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.
Gilbert Family Foundation Commits $11 Million to Combat Vision Loss in NF1 Patients
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 Vision Restoration Initiative aims to develop innovative therapies that either repair or replace the damaged optic nerve to restore vision to these patients.
Gilbert Family Foundation Announces $12 Million Gene Therapy Initiative to Address Underlying Cause of NF1
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.