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FDA Approves Leucovorin as First Drug for Rare Genetic Disorder

This approval follows previous claims of its potential use in treating autism symptoms.

Editorial Staff
1 min read
Updated 4 months ago

Updates

Update at 16:30 UTC on 2026-03-10

STAT reported The FDA approves leucovorin for a rare brain disorder while retreating from top officials’ earlier insistence that it could benefit many children with autism.

Sources: STAT

Update at 14:16 UTC on 2026-03-11

STAT reported The FDA's decision raises questions about the approval process and the implications for future drug evaluations.

Sources: STAT