Rare Disease Parents Create a New Model for Drug Development: Nicole Johnson, Co-Founder and Executive Director of FOXG1 Research Foundation

“As parents dedicated to getting a treatment for our children in their lifetimes, we have turned the rare disease drug development landscape upside down and created a new model,” says Nicole Johnson, co-founder and executive director of the FOXG1 Research Foundation.  That’s not an exaggeration, as the foundation is on track to make history as it begins patient clinical trials on a gene replacement therapy next year. The former TV news producer and media executive unexpectedly entered the world of patient advocacy and drug research after her daughter, Josie, was born with FOXG1, a genetic disorder which causes severe seizures and impedes normal movement, speech, and sleep among other problems. Johnson is also making an impact in another important dimension of the rare disease space in her efforts to educate parents, teachers, and students about disability inclusion through her Joyfully Josie book series and “Live Joyfully” education programs. Tune-in to this fascinating Year of the Zebra conversation with host Lindsey Smith to find out how the foundation is aiming to bring a drug to market in less than half the time and at a fraction of the cost than the industry standard, and how this model might impact research on other rare disorders. 

Mentioned in this episode:

FOXG1 Research Foundation
Joyfully Josie Book

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