Clinical and Translational Research at Cincinnati Children’s
The Tuberous Sclerosis Clinic at Cincinnati Children’s is active in many clinical, translational and basic science research studies aimed at improving care for children and adults with tuberous sclerosis. Clinical research is led by David Franz, MD, and Darcy Krueger, MD, PhD, in close collaboration with researchers in many departments at Cincinnati Children’s, the University of Cincinnati and around the world.
Studies include several human clinical trials aimed at determining the safety and effectiveness of mTOR inhibitors and other agents to treat subependymal giant cell astrocytoma (SEGA) tumors in the brain, angiomyolipomas (AMLs) in the kidneys and lymphangioleiomyomatosis (LAM) of the lungs. We are also investigating the use of these agents for the treatment of seizures, behavior problems and cognitive impairments that are frequently associated with TS. Additional projects aim to better understand how TS develops in individuals with genetic mutations in TSC1 or TSC2 and provide improved diagnostic tools and treatment approaches for TS.
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