Tuberous sclerosis medical therapy

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Tuberous sclerosis Microchapters


Patient Information


Historical Perspective


Differentiating Tuberous sclerosis from other Diseases

Epidemiology and Demographics


Natural History, Complications and Prognosis


Diagnostic Criteria

History and Symptoms

Physical Examination

Laboratory Findings


Chest X Ray



Echocardiography or Ultrasound

Other Imaging Findings

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Medical Therapy


Cost-Effectiveness of Therapy

Future or Investigational Therapies

Case Studies

Case #1

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]

Medical Therapy

Drug therapy for some of the manifestations of TSC is currently in the developmental stage.[1] For example, a 2008 study found that treatment with rapamycin rescued learning and memory deficits in a mouse model of tuberous sclerosis.[2] Community TSC is a distributed computing project to find drugs to treat TSC.[citation needed] The patients usually have relapse of symptoms in the clinical course. Unless any vital function is affected, life expectancy is good. Majority of patients will require some medications to control symptoms, e.g., anti-epileptics to control seizures. In 2010 everolimus was approved for the treatment of subependymal giant cell astrocytoma.

Other drugs used include:


  1. Yates JR (2006). "Tuberous sclerosis". Eur. J. Hum. Genet. 14 (10): 1065–73. doi:10.1038/sj.ejhg.5201625. PMID 16868562. Unknown parameter |month= ignored (help)
  2. Ehninger D, Han S, Shilyansky C; et al. (2008). "Reversal of learning deficits in a Tsc2+/− mouse model of tuberous sclerosis". Nat Med. 14 (8): 843–8. doi:10.1038/nm1788. PMC 2664098. PMID 18568033. Lay summaryScience News (23 June 2008).
  3. Tsao CY (2009). "Current trends in the treatment of infantile spasms". Neuropsychiatr Dis Treat. 5: 289–99. PMC 2695218. PMID 19557123.
  4. Simon D. Shorvon (2010). Handbook of Epilepsy Treatment. John Wiley and Sons. pp. 93–. ISBN 978-1-4051-9818-9. Retrieved 11 October 2010.