Fragile X Syndrome Medication

Updated: Aug 15, 2022
  • Author: Jennifer A Jewell, MD, MS; Chief Editor: Maria Descartes, MD  more...
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Medication Summary

The results of folic acid supplementation to curb the inattention and aggressiveness in prepubertal males are controversial; thus, folic acid supplementation is currently not the standard of care in fragile X syndrome. No effect has been observed in adults treated with folic acid.

Trials of medications, such as fenobam, that act as mGluR5 antagonists are underway. Excess mGluR5 signaling occurs when FMRP is decreased or absent. Therefore, downregulation of mGluR5 may improve outcomes in patients with fragile X syndrome. Lithium also inhibits mGluR5 signaling (as well as other pathways) and may benefit patients with fragile X syndrome.

Other trials with γ -aminobutyric acid agonists are underway.