Rare Disease Report

Managing Tuberous Sclerosis Complex is a Team Effort

APRIL 20, 2015

Karen Keough, MD, of Dell Children’s Medical Center in Austin, Texas, talks about managing patients with tuberous sclerosis complex (TSC).

TSC is a genetic disorder that causes nonmalignant tumors to form in many different organs—primarily in the brain, eyes, heart, kidney, skin and lungs. Symptoms vary with each patient, but the most common ones associated with TSC are seizures, developmental delay, intellectual disability, and/or autism.

Currently, there is no cure for TSC, although everolimus is approved to treat:
1) patients with subependymal giant cell astrocytoma associated with TSC who require therapeutic intervention but are not candidates for curative surgical resection, and
2) adults with renal angiomyolipoma and TSC not requiring immediate surgery.

For more information about TSC, visit www.tsalliance.org

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