How Do We Treat the Untreatable? SickKids VS Rare Disease
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 rema...
SickKids VS · SickKids Foundation
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Show Notes
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.)