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FDA Approves First Treatment For Patients With Cerebral Folate Transport Deficiency

The U.S. Food and Drug Administration has 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).

This action is part of the agency's efforts to accelerate cures and expand treatment options including for patients with serious and unmet needs. Leucovorin is the first treatment for the rare genetic condition of cerebral folate deficiency.

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by Joji Xavier

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