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Dr. Elizabeth A. Thiele

Elizabeth A. Thiele, MD, PhD

Dr. Elizabeth Thiele: It's been a very exciting time in the past few years in the world of tuberous sclerosis complex, because of what we have learned from the basic science advances. It's enabled us to think, really, for the first time, about specific treatments for TSC. Kind of historically, we've always treated the symptoms. We've treated the seizures. We've treated the kidney involvement with ways we would treat it. But there's never been a drug or a treatment, specifically at the underlying disorder. So, what the science has given us is tools to change that. And luckily, with what we've learned about the signal transduction pathways and cascades over the few years, we now have a drug, Rapamycin, that is in clinical trials. We don't know if this will be the drug for TS. But it's a great time of optimism, because it's a first drug that might treat TS. And we are already thinking, from what we have learned additionally, scientifically, of what might be the future drugs to try in trials, or what might be other mechanisms of treating the disorder, rather than treating the symptoms.