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RARECastAuthor: RARECast
RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy. Language: en Genres: Business Contact email: Get it Feed URL: Get it iTunes ID: Get it |
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How Parents Took Development of a Gene Therapy into Their Own Hands
Thursday, 23 April, 2026
Nicole Johnson and Nasha Fitter are both mothers of daughters with the ultra-rare neurodevelopmental condition FOXG1 syndrome, which currently has no approved disease-modifying therapies. The disorder causes profound developmental disabilities, epilepsy, motor and speech impairments, and multi-system challenges. The two mothers co-founded the FOXG1 Research Foundation to advance treatments for the condtion. Johnson and Fitter discuss how a parent-driven foundation became a virtual biotech capable of advancing a gene therapy into human clinical trials, how they prioritized translational work over academic projects that don’t move a therapy toward the clinic, and the lessons they’ve learned that can guide other rare disease communities.
