RareWays is a research directory. It helps you understand the science. It is not medical advice. Always discuss your care options with your healthcare team.
Coverage: 2020-01-01 onwards, with up to 300 results per query from PubMed and Europe PMC and separate limits on other sources. This is a selected index, not a complete literature search.
Updates are delayed or unverified. Check original sources for the latest information.
Your saved research and appointment questionsView recorded trial and retraction changes
Fragile X Syndrome: Research Summary
Printed from RareWays (rareways.org)
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Fragile X primary ovarian insufficiency
Boutrid N et al. (19 October 2026)
https://europepmc.org/search?query=Fragile%20X%20primary%20ovarian%20insufficiency
- 2.
A microglial basis for fragile X syndrome via impairment of FMRP-regulated lysosomal function.
Li Heng et al., Molecular psychiatry (17 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42892634/
- 3.
Variability in acoustic startle response and prepulse inhibition across adulthood in Fragile X messenger ribonucleoprotein 1 knockout mice.
Blandin Katherine J et al., Brain research (15 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42386069/
- 4.
Hyperexcitability in hippocampal CA1 pyramidal cells of Fmr1 knockout mice emerges during late postnatal development.
Luque M Ángeles et al., Neuroscience (15 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42595211/
- 5.
Atlas-based electrical source imaging for mouse EEG: Application to an Fmr1 knockout model.
Edmondson Alex D et al., NeuroImage (15 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42722311/
Clinical Trials : Australian Sites
Ask your doctor whether you or your child may be eligible for any of these trials. Each has its ID and full eligibility criteria at the link shown.
- 1.
Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40
NCT04823052 · WITHDRAWN · Phase 2 · Healx Limited
Ages 13 to 40
https://clinicaltrials.gov/study/NCT04823052
- 2.
Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome
NCT04977986 · Completed · Phase 3 · Harmony Biosciences Management, Inc.
Ages 3 to 29
https://clinicaltrials.gov/study/NCT04977986
- 3.
Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS
NCT03802799 · Active (not recruiting) · Phase 2 / Phase 3 · Zynerba Pharmaceuticals, Inc.
Ages 3 to 29
https://clinicaltrials.gov/study/NCT03802799
- 4.
Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX)
NCT03614663 · Completed · Phase 2 / Phase 3 · Zynerba Pharmaceuticals, Inc.
Ages 3 to 17
https://clinicaltrials.gov/study/NCT03614663
- 5.
Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)
NCT01433354 · TERMINATED · Phase 2 / Phase 3 · Novartis Pharmaceuticals
Ages 12 to 18
https://clinicaltrials.gov/study/NCT01433354
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Fragile X Syndrome
Fragile X syndrome is the most common inherited cause of intellectual disability and the leading single-gene cause of autism spectrum disorder, caused by expansion of a CGG repeat in the FMR1 gene. It affects males more severely than females. Research is focused on mGluR5 pathway modulation and gene reactivation strategies.
This overview is general information, not an individual medical assessment. Research is selected automatically and may include mismatches. How we select and explain research.
Most Recent Research
Common Questions
What is Fragile X Syndrome?
Fragile X syndrome is the most common inherited cause of intellectual disability and the leading single-gene cause of autism spectrum disorder, caused by expansion of a CGG repeat in the FMR1 gene. It affects males more severely than females. Research is focused on mGluR5 pathway modulation and gene reactivation strategies.
How many clinical trials are available for Fragile X Syndrome?
RareWays currently indexes 87 clinical trials for Fragile X Syndrome, of which 11 are actively recruiting. These counts cover indexed trials across countries, not just your country. An Australian site does not necessarily mean that site is recruiting. Check locations, eligibility and current availability with the study team.
Where does the research data for Fragile X Syndrome come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is refreshed automatically every day. All articles and trials link directly to their original sources.
Get research updates
Monthly email when new findings are published for Fragile X Syndrome.
Your email and selected disease are stored for these updates. Unsubscribe any time. Privacy and deletion.
This information is for general awareness only.
For guidance specific to your situation, please speak with your healthcare team.