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 Rett Syndrome
Rett syndrome is a rare genetic disease that causes developmental and nervous system problems, mostly in girls. It's related to autism spectrum disorder. Babies with Rett syndrome seem to grow and develop normally at first. Between 3 months and 3 years of age, though, they stop developing and even lose some skills. Symptoms include:
- Loss of speech
- Loss of hand movements such as grasping
- Compulsive movements such as hand wringing
- Balance problems
- Breathing problems
- Behavior problems
- Learning problems or intellectual disability
Rett syndrome has no cure. You can treat some of the symptoms with medicines, surgery, and physical and speech therapy. Most people with Rett syndrome live into middle age and beyond. They will usually need care throughout their lives.
It sits within Brain and Nerves and Genetics/Birth Defects.
The lead U.S. institute for this subject is the Eunice Kennedy Shriver National Institute of Child Health and Human Development.
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 Rett Syndrome
101 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
- Adapting a Psychosocial Support Intervention for the Rett Syndrome CommunityColorado State University
- A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett SyndromeHolland Bloorview Kids Rehabilitation Hospital
- Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)Rush University Medical Center
- Parent and Infant Inter(X)Action Intervention (PIXI)RTI International
- A Phase 3 Study of Fenfluramine Hydrochloride in Rett SyndromeUCB BIOSCIENCES, Inc.
- A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)Taysha Gene Therapies, Inc.
Research already completed
- Esketamine for the Treatment of Rett SyndromeChildren's Hospital of Fudan University
- Osteoporosis in RETT SyndromeAssistance Publique - Hôpitaux de Paris
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
- Rett SyndromeNational Institute of Neurological Disorders and Stroke
- Rett SyndromeMayo Foundation for Medical Education and Research
- Rett Syndrome: OverviewEunice Kennedy Shriver National Institute of Child Health and Human Development
Symptoms
- What Are the Symptoms of Rett Syndrome?Eunice Kennedy Shriver National Institute of Child Health and Human Development
Diagnosis and Tests
- How Do Health Care Providers Diagnose Rett Syndrome?Eunice Kennedy Shriver National Institute of Child Health and Human Development
Treatments and Therapies
- What Are the Treatments for Rett Syndrome?Eunice Kennedy Shriver National Institute of Child Health and Human Development
Genetics
- Rett syndrome: MedlinePlus GeneticsNational Library of Medicine
- What Causes Rett Syndrome?Eunice Kennedy Shriver National Institute of Child Health and Human Development
Clinical Trials
- ClinicalTrials.gov: Rett SyndromeNational Institutes of Health
Medical subject headings
Clinicians and researchers index this subject under Rett Syndrome. These are the terms to use when searching medical literature.
This page is built from the MedlinePlus health topic record for Rett Syndrome, 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.