Building Progress in Friedreich’s Ataxia - Advocacy, Science, and the FALCON Trial - Thomas Hamilton скачать в хорошем качестве

Building Progress in Friedreich’s Ataxia - Advocacy, Science, and the FALCON Trial - Thomas Hamilton

Thomas Hamilton is a Board Director Friedreich's Ataxia Research Alliance ‪@CureFA‬ ( FARA - https://www.curefa.org/ ), Co-Founder of the CureFA Foundation ( https://curefafoundation.org/ ), which supports diverse and innovative research strategies aimed at curing the disease, and the Founder of FA212, LLC, an organization pursuing a novel gene therapy strategy designed to address both central nervous system and systemic manifestations of FA. Thomas' journey into rare disease advocacy began not in a laboratory, but at home when in 2013, his nine-year-old daughter Annie was diagnosed with Friedreich’s Ataxia — a rare, progressive neuromuscular disorder that affects balance and coordination and can lead to scoliosis, diabetes, sensory loss, and serious cardiac complications. Friedreich's ataxia (FA) is a rare, inherited, progressive neurodegenerative disorder caused by a mutation in the FXN gene, which leads to a deficiency of the frataxin protein. Like many families facing a rare diagnosis, the Hamilton family was suddenly thrust into a world of uncertainty, limited treatment options, and an urgent need for answers. Rather than accept the status quo, Thomas made the life-altering decision to step away from his professional career and dedicate himself fully to accelerating therapeutic progress for FA. Thomas joined the executive leadership of the Friedreich's Ataxia Research Alliance (FARA), where he remains an active board member, and co-founded the CureFA Foundation to support diverse and innovative research strategies aimed at curing the disease. In 2014, working alongside family members and clinical partners, Tom helped establish an FA Center of Excellence at Children's Hospital of Philadelphia ( ‪@ChildrensHospPhila‬https://www.research.chop.edu/friedre... ) in collaboration with University of Pennsylvania — creating a dedicated clinical and research hub for patients and scientists focused exclusively on Friedreich’s Ataxia. Driven by the belief that bold approaches were needed, Thomas also founded FA212, LLC to pursue a novel gene therapy strategy designed to address both central nervous system and systemic manifestations of FA. That program has evolved into SGT-212, now being advanced by ‪@SolidBiosciences‬ ( https://www.solidbio.com/our-science/... ). The therapy has entered human clinical evaluation through the FALCON study ( https://clinicaltrials.gov/study/NCT0... ), with the first participant recently dosed at Ohio State University Wexner Medical Center — marking a milestone not only for the Hamilton family but for the broader FA community. Today, Annie is 19 and a student at University of Notre Dame, living with FA while witnessing firsthand the progress her family helped catalyze. Thomas and Annie’s story represents the growing power of parent-driven innovation in rare disease — where urgency, collaboration, and perseverance can reshape the therapeutic landscape. #FriedreichsAtaxia #RareDisease #GeneTherapy #SGT212 #FALCONTrial #RareDiseaseAdvocacy #PatientAdvocate #RareDiseaseResearch #BiotechInnovation #MitochondrialDisease #Ataxia #PrecisionMedicine #ClinicalTrials #HopeInScience #CureFA #FARA #NeurodegenerativeDisease #MedicalBreakthrough #ParentAdvocate #ProgressPotentialPossibilities