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🔴 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).

View FDA Announcement
Read the original at FDA
FDA ApprovalTreatment

Related conditions

Neurodegenerative syndrome due to cerebral folate transport deficiency

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