About five months after U.S. Health Secretary Robert F. Kennedy Jr. touted leucovorin as “an exciting therapy that may benefit large numbers of children who suffer from autism” during a White House press conference, the FDA has approved the decades-old drug for a rare genetic condition with “autistic features” that represents an ultrarare subset of autism patients.
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- FDA approves leucovorin for ultrarare cerebral folate deficiency subset without clinical trial
FDA approves leucovorin for ultrarare cerebral folate deficiency subset without clinical trial
- March 10, 2026
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