Thrilled to share my latest @ProgressPotent1 episode: "Building Progress in Friedreich’s Ataxia - Advocacy, Science, and the FALCON Trial" with @tehamilton211
From a dad's diagnosis-driven mission in 2013 to co-founding CUREFA FOUNDATION INC, leading Friedreich's Ataxia Research Alliance (FARA) efforts, establishing a @ChildrensPhila Center of Excellence, and pioneering gene therapy via FA212 → SGT-212.
Huge milestone: The first participant was dosed in @Solid_Bio 🚀
Phase 1b FALCON trial in Jan 2026 - targeting both CNS & systemic FA manifestations with dual-route delivery!
Thomas & daughter Annie (now 19, at @NotreDame ) embody parent-powered innovation turning urgency into hope for the FA community.
Watch now for this story of perseverance, collaboration, and accelerating cures!
#FriedreichsAtaxia #RareDisease #GeneTherapy #SGT212 #FALCONTrial #RareDiseaseAdvocacy #PatientAdvocate #CureFA #FARA #BiotechInnovation #MitochondrialDisease #PrecisionMedicine #ClinicalTrials #HopeInScience #ParentAdvocate @FINNPartners @PennMedicine @OhioState
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