Against All Odds: Finding a Treatment for SPG50
Description
We can think of no better way to kick off Season 3. In a deeply personal and moving episode, two patient advocates share their stories of working tirelessly to deliver a safe and effective gene therapy for children suffering from spastic paraplegia 50 (SPG50).
After his infant son received a diagnosis of this ultrarare neurodegenerative disease in 2017, Terry Pirovolakis began a daunting four-year journey to find a breakthrough treatment that would help Michael and other affected children. We also hear from Samuela Bellini, who wants to bring this therapy to children in Italy. Their achievements, and reflections, are an inspiration for our industry.
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