State of Digital Clinical Trials Podcast	 cover image

State of Digital Clinical Trials Podcast

Against All Odds: Finding a Treatment for SPG50

Sep 25, 2024
29:20

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.


Subscribe to State of Digital Clinical Trials, a Veeva podcast, to get new episodes as soon as they are released.


Remember Everything You Learn from Podcasts

Save insights instantly, chat with episodes, and build lasting knowledge - all powered by AI.
App store bannerPlay store banner