Also called: TSC · Tuberous sclerosis complex
Important: This page is general information, not medical advice. It does not recommend or prescribe any treatment. Always consult your doctor or pharmacist before starting, stopping or changing any medicine.
About Tuberous Sclerosis
Tuberous sclerosis is a rare genetic disease that causes benign tumors to grow in the brain and other organs. Symptoms vary, depending on where the tumors grow. They could include:
- Skin problems, such as light patches and thickened skin
- Seizures
- Behavior problems
- Intellectual disabilities
- Kidney problems
Some people have signs of tuberous sclerosis at birth. In others it can take time for the symptoms to develop. The disease can be mild, or it can cause severe disabilities. In rare cases, tumors in vital organs or other symptoms can be life-threatening.
Tuberous sclerosis has no cure, but treatments can help symptoms. Options include medicines, educational and occupational therapy, surgery, or surgery to treat specific complications.
It sits within Brain and Nerves and Genetics/Birth Defects.
The lead U.S. institute for this subject is the National Institute of Neurological Disorders and Stroke.
When to speak to someone. Reading about a topic cannot tell you whether it applies to you. Speak to a doctor or pharmacist if symptoms are severe, sudden, getting worse, or simply not going away — and seek emergency care immediately for chest pain, breathing difficulty, sudden weakness or confusion, or a serious allergic reaction.
Research on Tuberous Sclerosis
129 studies areregistered for this condition on ClinicalTrials.gov, the U.S. National Library of Medicine's public registry. A selection is below — the links go straight to the registry entry, not to a summary of ours.
What a registered study does and does not mean. Registration means a study exists and has been declared publicly. It says nothing about whether the treatment being tested works, is safe, or is available to you. Many studies find no benefit — that is what research is for. Never seek out an experimental treatment on the strength of a registry entry alone.
Research currently under way
- Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral SolutionJazz Pharmaceuticals
- Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental DisordersHolland Bloorview Kids Rehabilitation Hospital
- Role of Genetic Factors in the Development of Lung DiseaseNational Heart, Lung, and Blood Institute (NHLBI)
- Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)Rush University Medical Center
- Parent and Infant Inter(X)Action Intervention (PIXI)RTI International
- Assessing Changes in Multi-parametric MRI in MS Patients Taking Clemastine Fumarate as a Myelin Repair TherapyUniversity of California, San Francisco
Research already completed
- Early Behavioral Intervention to Improve Social Communication Function in Infants With Tuberous Sclerosis ComplexUniversity of California, Los Angeles
- Gait in Rare DiseasesUniversiteit Antwerpen
- A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis ComplexJazz Pharmaceuticals
Study data from ClinicalTrials.gov, a service of the U.S. National Library of Medicine. Retrieved 2026-07-30. Registry entries are supplied by study sponsors and investigators; ClinicalTrials.gov does not verify their scientific validity. PocketsInfo is not affiliated with, and not endorsed by, the NLM or the NIH.
Where to read more
Rather than paraphrase, we point you to the primary sources. Every link below is to a government health agency, a national institute or a recognised medical body — grouped by what you are trying to find out.
Start Here
- Tuberous Sclerosis ComplexNational Institute of Neurological Disorders and Stroke
Genetics
- Tuberous sclerosis complex: MedlinePlus GeneticsNational Library of Medicine
Clinical Trials
- ClinicalTrials.gov: Tuberous SclerosisNational Institutes of Health
Medical subject headings
Clinicians and researchers index this subject under Tuberous Sclerosis. These are the terms to use when searching medical literature.
This page is built from the MedlinePlus health topic record for Tuberous Sclerosis, a service of the U.S. National Library of Medicine (NLM), National Institutes of Health. Information is from MedlinePlus.gov. PocketsInfo is not affiliated with, and is not endorsed by, the NLM or the NIH. Retrieved 30 July 2026.