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Behavioural difficulties in fragile X syndrome: current pharmacological options and potential future developments
Andrew C Stanfield1, Andrew G McKechanie1
1Patrick Wild Centre, Centre for Clinical Brain Sciences, University of Edinburgh, Edinburgh, UK.
Expert Review of Neurotherapeutics
|June 4, 2025
Summary
Fragile X Syndrome (FXS) management involves non-pharmacological approaches and targeted treatments. Novel medications and genetic therapies are in development for FXS behavioral difficulties.
Area of Science:
- Neuroscience
- Genetics
- Pharmacology
Background:
- Fragile X Syndrome (FXS) is a leading inherited cause of intellectual disability and autism spectrum disorders.
- FXS is characterized by hyperarousal, anxiety, and challenging behaviors like aggression and self-injury.
Purpose of the Study:
- To review the physical, cognitive, and behavioral phenotype of FXS.
- To evaluate pharmacological interventions for FXS-related behavioral issues, including symptom-based and targeted treatments.
- To discuss emerging targeted therapies for FXS.
Main Methods:
- Narrative review of existing literature.
- Analysis of evidence for pharmacological interventions.
- Assessment of clinical trial data for novel treatments.
Main Results:
- Non-pharmacological interventions are first-line for FXS behavioral difficulties.
- Limited evidence exists for current pharmacological approaches, often based on small studies.
- Several novel targeted treatments are in late-stage development, with genetic therapies preclinical.
Conclusions:
- Management of anxiety and ADHD is crucial before considering antipsychotics for severe behaviors.
- Repurposed targeted treatments require careful monitoring due to limited trial data.
- Future FXS treatment may involve novel medications and genetic therapies.
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