🔴 BreakingNewsFDATuesday, March 10, 2026 · March 10, 2026
FDA Approves First Treatment for Patients with Cerebral Folate Transport Deficiency
WHY IT MATTERS
FDA announcement relevant to rare disease patients and caregivers.
The U.S. Food and Drug Administration today approved expanded use of Wellcovorin (leucovorin calcium) tablets for the treatment of cerebral folate deficiency in adult and pediatric patients who have a confirmed variant in the folate receptor 1 gene (CFD-FOLR1).
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