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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 about 1 month ago
Summary
Summary
- Leucovorin is now the first approved drug for a specific rare genetic disorder.
- The approval comes after months of discussions regarding its efficacy for autism.
- The Trump administration previously promoted leucovorin for broader autism treatment.
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