A New Approach May Help Patients with Fragile X Autism

Baclofen, a GABA_B agonist, seems to help socialization, especially among the most severely afflicted individuals.

In preclinical research on the pathways affected by the fragile X mutation (>200 CGG repeats on FMR1), researchers found increased glutamate activity at the mGlu5 receptor (JW Psychiatry Feb 25 2008). Now, investigators have examined the effect of inhibiting excessive glutamate production by increasing gamma-aminobutyric acid (GABA) B activity with arbaclofen, the R-enantiomer of racemic baclofen, a GABA_B receptor agonist usually prescribed to alleviate spasticity. This crossover study was supported by the manufacturer.

The 63 participants (age range, 6–40 years) were randomized to receive titrated active drug or placebo for 4 weeks; crossover to the other arm occurred after a 2- to 3-week taper and washout. More than 85% of participants con…

Reviewing Author

Barbara Geller, MD

Disclosures

Nothing to disclose

Barbara Geller, MD

Disclosures

Nothing to disclose