Background

Many individuals with FXS experience behavioural problems such as anxiety, hyperarousal (heightened stress or sensory reactivity), agitation, aggression, and self-injurious behaviour.
These difficulties can significantly affect quality of life, safety, and social functioning. Managing these behaviours is a major focus in FXS care, yet effective drug treatments remain limited.

Study Methods

This article is a narrative review that summarizes current evidence and expert opinion on the pharmacological (drug-based) management of behavioural problems in people with Fragile X Syndrome.
We reviewed clinical studies, case reports, and ongoing trials to evaluate which medications are used, what evidence supports their use, and what new therapies are emerging. We also discussed the role of non-drug (non-pharmacological) strategies and future directions, including targeted and genetic treatments.

Key Findings

• Non-drug interventions come first: Behavioural and environmental approaches should be the first line of management before considering medications.
• Limited evidence for current drugs: While medications such as antipsychotics, antidepressants, and stimulants are sometimes used to manage anxiety, hyperactivity, or aggression in FXS, there is limited high-quality research specific to this condition.
• Treating underlying symptoms helps: Addressing anxiety, ADHD, or other contributing issues may reduce secondary behaviours like aggression or self-injury.
• Emerging treatments: Several drugs originally developed for other conditions (“repurposed targeted treatments”) are being studied for their potential to correct biological abnormalities in FXS. However, most studies so far are small or early-phase.
• Future directions: Novel medications and genetic therapies are in development and may offer more precise, effective treatments in the coming years.

Conclusion

Managing behavioural problems in Fragile X Syndrome requires a careful, stepwise approach.
Non-drug strategies remain essential, while current medications can help some individuals but must be used cautiously due to limited evidence and potential side effects.
Research into targeted and genetic therapies is advancing and holds promise for more effective, biologically based treatments in the future.