FOXG1 is a rare and severe neurological disorder caused by a deficiency in the FOXG1 protein expression. There are fewer than 1,000 known cases of FOXG1 worldwide, mostly children under the age of 10. There is no cure, and Big Pharma does not prioritize research for rare diseases.

The aim of this project is to find a treatment for FOXG1, providing hope and advocacy for the FOXG1 community and rare diseases more broadly, by supporting independent research through strategic partnerships with leading universities and biotech companies.

Using FDA-approved methods, current research teams are exploring multiple ways to normalize FOXG1 protein expression. They have made incredible advancements, including the development of a novel gene therapy drug (at UMass Medical School) and the creation of FOXG1 impaired mice (at the McLaughlin Research Institute in Montana). In parallel, they are working to understand the underlying mechanisms of this disease by using novel technology at Washington University in St. Louis.

Goal

• Support current gene therapy efforts by measuring the efficacy of this new gene therapy drug through novel MERFISH technology at Washington University in St. Louis.

Activities

• Support research experiments and analysis work to test and measure the efficacy of this new gene therapy drug in FOXG1-impaired mice, to be conducted at Washington University in St. Louis.

Myriad Canada is working with Believe in a Cure on this project, a nonprofit whose mission is to support scientific research with the ultimate goal of finding a treatment or cure for FOXG1 syndrome.

Believe in a Cure’s mission is to find and fund the best scientists in the world to develop therapies that improve the lives of FOXG1 patients ; to collaborate with others who help them do so ; and to use the links between FOXG1 and diseases like Alzheimer’s, Schizophrenia, and Autism Spectrum Disorder to share knowledge and help as many people as possible.

Believe in a Cure has established partnerships with the National Institute of Health, leading universities, and biotech companies across the U.S., Europe, and Asia. It has assembled a Scientific Advisory Panel, recruited over 250+ scientists to the field, and commissioned research teams operating within the U.S. and internationally. In addition to supporting research programs, Believe in a Cure provides support and resources to rare disease families.