Medication Guide for Fragile X Syndrome
This guide is intended to help parents and caregivers who are seeking or considering medication to treat the behavioral and psychiatric challenges of Fragile X syndrome. While some treatment approaches have evolved since its publication, the insights in this manual continue to offer a valuable foundation for understanding medications and treatment strategies.
It is not a substitute for medical advice. Instead, it supports informed conversations with qualified healthcare providers. In an ideal world, every physician would have extensive training in Fragile X and the time to educate each family. Since that’s not always the case, this guide helps bridge the gap by offering plain-language background on the psychopharmacology of Fragile X.
Disclaimer
This guide is not a medical textbook and cannot take the place of a qualified physician.
It is intended to serve as background information to help parents, caretakers and others communicate with their physicians regarding medications. The medications described in this guide are to be used only under the supervision of a qualified physician; almost all are available only by prescription.

Published March 2009
This is Version 5 of the Fragile X Medication Guide. While it is not actively updated, it remains available as a historical reference.
Written by
Michael Tranfaglia, MD
Medical Director, Treasurer, Co-Founder
Dr. Michael Tranfaglia is Medical Director and Chief Scientific Officer of FRAXA Research Foundation, coordinating the Foundation’s research strategy and working with university and industry scientists to develop new therapeutic agents for Fragile X. He has a BA in Biology from Harvard University and an MD from the University of North Carolina at Chapel Hill. His son Andy has Fragile X syndrome.
Explore More on Fragile X Treatment
Shionogi’s EXPERIENCE Phase 3 Clinical Trial of Zatolmilast in Fragile X Syndrome
Shionogi’s EXPERIENCE clinical trials for Fragile X syndrome are nearing completion. Enrollment for the adult trial (EXPERIENCE-301) is now closed, while the adolescent trial (EXPERIENCE-204) is in its final phase. Learn more about the study, FRAXA’s role, and the open-label extension.
Read More »NPR Spotlights Zatolmilast: A Potential Breakthrough for Fragile X Syndrome
NPR spotlights zatolmilast, a promising drug offering new hope for individuals with Fragile X syndrome. Families report life-changing improvements in anxiety, communication, and independence.
Read More »NPR Feature – A Fragile X Treatment May Be On The Horizon
With a promising new treatment on the horizon, NPR reflects back at the past 10 years, providing a glimpse into both the science and research of Fragile X through the eyes of key researchers, in addition to the lives of this family, their quest for a cure, and what that may look like for their son, Andy.
Read More »