Deirdre McCarthy: The Long Pursuit of a Fragile X Syndrome Treatment

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There is currently no approved treatment for fragile X syndrome.

There remains no approved treatments for fragile X syndrome (FXS), the leading inherited cause of intellectual disabilities and autism spectrum disorder (ASD) worldwide.

However, preclinical models show kappa opioid receptor antagonists have therapeutic potential to help treat the disorder.

In research presented during the 2021 American Professional Society of ADHD and Related Disorders (APSARD) Annual Virtual Meeting, a team of researchers presented data showing kappa opioid receptor antagonists could eventually be a much needed viable treatment for fragile X syndrome.

In an interview with HCPLive®, Deirdre McCarthy, Department of Biomedical Sciences, Florida State University College of Medicine, explained the promising results and what the plans are to further study this treatment in larger human trials.

McCarthy discussed why it is so difficult to find a suitable treatment for fragile X syndrome and how this treatment is surprisingly promising for this disease.

McCarthy said kappa opioid receptor antagonists have been used in preclinical models as a treatment for addiction disorders and pain, which led the researchers to the belief that it could be a potential therapy for fragile X syndrome.

These drugs can modulate therapeutic transmitters in the brain.

McCarthy said there are also many different drugs that are showing promise in preclinical models.