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RARECast
RARECast
577 episodes
4 days ago

RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.

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Business
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All content for RARECast is the property of RARECast and is served directly from their servers with no modification, redirects, or rehosting. The podcast is not affiliated with or endorsed by Podjoint in any way.

RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.

Show more...
Business
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Transforming TSC Epilepsy with a Precision Therapy
RARECast
25 minutes 6 seconds
5 days ago
Transforming TSC Epilepsy with a Precision Therapy
Tuberous sclerosis complex is a rare genetic condition caused by changes in the TSC1 or TSC2 gene that over-activate a key growth-control pathway, known as mTOR. This leads to seizures that often don’t respond to existing medicines and to noncancerous tumors in organs such as the brain, kidneys, lungs, and skin. In fact, more than 65 percent of people with TSC-related epilepsy still have seizures despite available treatments. Though mTOR inhibitors can help some patients, their benefits are limited because of side effects due to their lack of specificity. Aeovian Pharmaceuticals is developing an experimental therapy that precisely targets the overactive part of this pathway and avoids the part thought to cause many side effects. We spoke to Allison Hulme, CEO of Aeovian Pharmaceuticals, about tuberous sclerosis complex, the problems with existing therapies for seizures related to the condition, and the opportunity for its next-generation, selective mTOR inhibitors to treat tuberous sclerosis complex and beyond.    
RARECast

RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.