Often when a child is diagnosed with FXS, parents call the NFXF and ask us about medication that might help treat or cure their child. There are many different aspects of medical treatment for FXS. These include treatment of behavioral/mental health issues, treatment for medically associated issues such as seizures, and medication treatments being investigated in “clinical trials” to directly impact the effects of the FMR1 mutation that causes FXS.Here we will describe some of the classes of medications that may be prescribed by physicians. Please note that this discussion is for general informational purposes only, and that the NFXF does not evaluate, recommend or endorse specific medications. Please consult your individual medical providers to address your particular circumstances.
Medications for Behavioral/Mental Health Issues
Your child’s physician can consider various classes of medications to treat behavioral and mental health conditions associated with Fragile X syndrome. Some childen with FXS benefit from medications that treat ADD, ADHD and other attention disorders. Other children who experience general anxiety, social anxiety, OCD and other perseverative disorders may benefit from different types of anti-anxiety medications. Parents often have to work with their child’s doctor in trying more than one medication before finding the best fit for their child. For children facing more significant psychiatric challenges, a number of available “anti-psychotic” medications may be of value.
Treatment of Related Health Issues
Children with FXS have a higher incidence of various medical/health issues that often require medications. Prominent among these are anti-seizure medications and antibiotics for recurrent ear and sinus infections. Some babies with FXS also have problems with gastrointestinal reflux and may be prescribed medication to help treat their symptoms.
Clinical Trials for Medications
Families often read or hear about medications to treat FXS being developed or actively used in research trials. Many of these medications are under investigation for their potential to treat the symptoms of the condition, particularly its cognitive and behavioral aspects. It is therefore important to understand that clinical trial medications are not available through your health care provider.
A clinical trial is a study in which the participants are given the drug for a specific period of time and are carefully monitored for both positive and negative responses. The trials proceed through different phases to assess appropriate dosages, possible toxicity, clinical outcomes, etc. Clinical trials usually occur within a medical center, often where there is a Fragile X Clinics and a researcher (a “P.I.” or primary investigator) who heads up the study. We have more information about clinical trials and possible opportunities to participate on our Opportunities for Families page.