Rare Disease Report

Tuberous Sclerosis Alliance

OCTOBER 07, 2013

At the 2013 DIA NORD Conference on Rare Diseases & Orphan Drugs, we had the privilege of talking to Kari Luther Rosbeck, President and CEO of the Tuberous Sclerosis Alliance.  In this interview, Kari talks about the origins of the Alliance.

Tuberous sclerosis complex (TSC) is a genetic disorder that causes non-malignant tumors to form in many different organs, primarily in the brain, eyes, heart, kidney, skin and lungs. Symptoms vary but the ones most commonly associated TSC are the central ones including, seizures, developmental delay, intellectual disability and autism.  At present there is no cure for TSC however Afinitor (everolimus) is approved to treat

  • Patients with subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis who require therapeutic intervention but are not candidates for curative surgical resection
  • Treatment of adults with renal angiomyolipoma and tuberous sclerosis complex (TSC) not requiring immediate surgery.

For more information about the Alliance, visit www.tsalliance.org

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