A top podcast for healthcare leaders, with over one million downloads, Radio Advisory is your weekly download on how to untangle the industry's most pressing challenges to help leaders like you make the best business decisions for your organization. From unpacking major trends in care delivery—like site-of-care shifts and the rise of high-cost drugs—to demystifying stakeholder dynamics, to shining a spotlight on priorities that may get overlooked, we're here to help. Our hosts and seasoned r ...
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Summit Pursues A Different Approach to Treating Duchenne
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Manage episode 181478086 series 60790
Content provided by RARECast. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by RARECast 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.
Duchenne Muscular Dystrophy, a rare genetic disease, causes progressive muscle wasting that slowly robs people of abilities and leads to death. In recent years, much attention has focused on the use of antisense oligonucleotides to bypass defective portions of the exon that codes for the dystrophin gene to restore its production. Dystrophin is a protein that is essential to healthy muscle. Summit Therapeutics is taking a different approach. Instead of restoring dystrophin, Summit is developing a drug that activate utrophin, a related protein that serves a similar function to dystrophin during fetal development, but then shuts off. We spoke to Glyn Edwards, CEO of Summit, about the company’s utrophin activator ezutromid, its licensing deal with Sarepta Therapeutics, and why, unlike the exon-skipping drugs that target specific subpopulaitons of Duchenne patients, ezutromid could provide benefits to patients broadly.
…
continue reading
510 episodes
MP3•Episode home
Manage episode 181478086 series 60790
Content provided by RARECast. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by RARECast 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.
Duchenne Muscular Dystrophy, a rare genetic disease, causes progressive muscle wasting that slowly robs people of abilities and leads to death. In recent years, much attention has focused on the use of antisense oligonucleotides to bypass defective portions of the exon that codes for the dystrophin gene to restore its production. Dystrophin is a protein that is essential to healthy muscle. Summit Therapeutics is taking a different approach. Instead of restoring dystrophin, Summit is developing a drug that activate utrophin, a related protein that serves a similar function to dystrophin during fetal development, but then shuts off. We spoke to Glyn Edwards, CEO of Summit, about the company’s utrophin activator ezutromid, its licensing deal with Sarepta Therapeutics, and why, unlike the exon-skipping drugs that target specific subpopulaitons of Duchenne patients, ezutromid could provide benefits to patients broadly.
…
continue reading
510 episodes
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