US FDA Approves First Treatment for Patients with Cerebral Folate Transport Deficiency
United States, March 11 -- The U.S. Food and Drug Administration 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 reflects the agency's commitment to accelerating cures and expanding treatment options including for patients with serious and unmet needs. Leucovorin is the first treatment for the rare genetic condition of cerebral folate deficiency.The approval was based on a systematic review of the published literature on the topic, including published case reports with patient-level information, as well as mechanistic data. Cerebral folate deficiency is a neurol...
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