Rett Syndrome
Recent clinical, regulatory, research and industry developments relating to this disease.
Emerging therapies for childhood-onset movement disorders.
Reversal of neurological deficits by painless nerve growth factor in a mouse model of Rett syndrome.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
- 2 clinical trials expected to report results, the earliest in Q4 2026.
Clinical MilestonesViewHide
- 2026-05-01Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome Using an "N of 1" Study DesignResults expected Q4 2026
- 2026-03-27A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Phase 3 Study of Bionetide for the Treatment of Girls and Women With Rett SyndromeResults expected Q3 2027
- 2026-06-30Repurposing Mirtazapine in Rett Syndrome: a Multicentric Open Label Phase II StudyPrimary completion
- 2025-12-01Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)Primary completion
- 2026-06-30ClinicalRepurposing Mirtazapine in Rett Syndrome: a Multicentric Open Label Phase II StudyPrimary completion
- 2026-05-01ClinicalRett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome Using an "N of 1" Study DesignResults expected Q4 2026
- 2026-03-27ClinicalA Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Phase 3 Study of Bionetide for the Treatment of Girls and Women With Rett SyndromeResults expected Q3 2027
- 2025-12-01ClinicalAssessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)Primary completion
Clinical trials
The current development programme across all trial phases.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Major themes6
- Rett Syndrome3
- CRISPR-Cas Systems1
- Gene Editing1
- Movement Disorders1
- Neuronal Plasticity1
- Synapses1
Leading journals4
- Advanced science (Weinheim, Baden-Wurttemberg, Germany)1
- Biomolecules1
- Brain : a journal of neurology1
- Current opinion in pediatrics1
Leading researchers8
- Borgonovo G1
- Buvinic S1
- Calvello M1
- Capsoni S1
- Cattaneo A1
- Cho HY1
- Choi JW1
- Di Caprio M1
Affiliations (unnormalised)6
- Bio@SNS Laboratory of Biology1
- F.M. Kirby Neurobiology Center1
- Institute of Neuroscience1
- Kookmin University1
- Neuromuscular Studies Laboratory (NeSt Lab)1
- Rita Levi-Montalcini European Brain Research Institute (EBRI)1
Disease biology
Key proteins & gene products studied in this disease. Number shows shared papers.
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Reference
Authoritative identity, definition & identifiers.
An inherited neurological developmental disorder that is associated with X-LINKED INHERITANCE and may be lethal in utero to hemizygous males. The affected female is normal until the age of 6-25 months when progressive loss of voluntary control of hand movements and communication skills; ATAXIA; SEIZURES; autistic behavior; intermittent HYPERVENTILATION; and HYPERAMMONEMIA appear. (From Menkes, Textbook of Child Neurology, 5th ed, p199)
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.