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Children’s Hospital Boston scientists have initiated a clinical trial to test a potential treatment for Rett Syndrome, known to cause autism in girls.

The three-year trial will test mecasermin, a drug candidate that the U.S. Food and Drug Administration has already approved for IGF-1 deficiency-linked shortness. Forty girls diagnosed with Rett syndrome will participate in the trial, which will randomly assign placebo or mecasermin. The research team will study the results based on neurodevelopment and cardiorespiratory outcomes.

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