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FDA Weighs Leucovorin for Autism Treatment in Children

10/2/2025

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Summary

Story summary

The U.S. Food and Drug Administration (FDA) is considering leucovorin to treat autism in children with cerebral folate deficiency (CFD).
It may improve language and social skills for some children, but benefits are not universal.
Experts urge more research. Side effects may include irritability and hyperactivity, usually mild. Folate testing is specialized and may not be covered by insurance.

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Story summary

The U.S. Food and Drug Administration (FDA) is considering leucovorin to treat autism in children with cerebral folate deficiency (CFD).
It may improve language and social skills for some children, but benefits are not universal.
Experts urge more research. Side effects may include irritability and hyperactivity, usually mild. Folate testing is specialized and may not be covered by insurance.