Rare As One Partner
FOXG1 Research Foundation
Curing diseases is not science fiction. In the age of exponential advances in genetic science, the founders of the FOXG1 Research Foundation, Nasha Fitter and Nicole Johnson, knew that to save their children, they would have to create a strategic foundation and run it like a business.
This meant innovation—disrupting the models that did not work and pioneering new methods to advance drug development. This meant collaboration—connecting to the global community of FOXG1 parents, and launching affiliate chapters worldwide, and developing a consortium of the leading scientists to work together. This meant organization—mapping the path to a cure and gathering the assets and data needed along this path.
FOXG1 syndrome is a rare neurodevelopmental disorder caused by a mutation in the FOXG1 gene, which is one of the first and most critical genes in brain development. Most children with FOXG1 syndrome are physically and cognitively disabled and also have epilepsy.
The FOXG1 Research Foundation has developed models (animal and cellular) to represent the gamut of all mutation types and is testing FDA-approved drugs, antisense therapies, and gene therapies on these models.
The Chan Zuckerberg Initiative supports the FRF’s development of an innovative new digital natural history study using machine learning. The study is designed to break down the barriers to participation and accessibility in the current natural history study model, and also aims to accelerate rare disease drug development.