))
How Do We Treat the Untreatable? SickKids VS Rare Disease
Manage episode 365531505 series 3277297
Content provided by SickKids Foundation. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by SickKids Foundation or their podcast platform partner. If you believe someone is using your copyrighted work without your permission, you can follow the process outlined here https://player.fm/legal.
Five-year-old Michael is one of less than 100 people in the world with SPG50, an ultrarare genetic disease. In March 2022, a SickKids team dosed Michael with the first-ever gene therapy for SPG50 in an unprecedented clinical trial. We tell the remarkable story of what it took to deliver a customized drug for Michael’s particular genetic variant, and what it means for SickKids’ vision of precision medicine.
(For bonus content or to advance similar research, please visit SickKidsFoundation.com/podcast. The SickKids VS Podcast is generously sponsored by CIBC and edited by Quill.)
37 episodes
Share
