Against All Odds: Finding a Treatment for SPG50

episode

Unblinded: A Clinical Trial Podcast not yet transcribed

▲ 0

Transcript

This episode hasn't been transcribed yet Upvotes decide what gets transcribed next — 0 so far. Sign in to upvote

Related

recent transcribed audio

AWP: Beyond the Exam Room: Purpose, Myopia, and Population Health 54 min · 17 Sep 2026 · EyeCode Media

Essentials: How to Assess & Improve All Aspects of Your Fitness | Dr. Andy Galpin 35 min · 17 Sep 2026 · Huberman Lab

#502 – Psychiatry, Insane Asylums, Mental Illness, ECT, Lobotomies, Freud & Jung 3h 43m · 17 Sep 2026 · Lex Fridman Podcast

Your partner wants profit. You want freedom. Now what? 22 min · 16 Sep 2026 · The Optometry Success Podcast

Board of County Supervisors Meeting - Work Session - Sep 15, 2026 3h 17m · 15 Sep 2026 · Prince William County Board of Supervisors

Finance and Budget Committee - Sep 15, 2026 1h 43m · 15 Sep 2026 · Prince William County Finance and Budget Committee

Report any issue

Source

Recorded25 Sep 2024

Publisherfeed.podbean.com ↗

Handlerec:14731158

Show notes

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.

Ask this episode

“What did the council decide on item 42?” — answered with the speaker and the moment.

Sign in