🔴 BreakingDrug approvalRSSApr 3
FDA Approves First Treatment for Patients with Cerebral Folate Transport Deficiency
The FDA has approved a new use for a medicine called Wellcovorin (leucovorin calcium) to treat a rare condition called cerebral folate deficiency. This condition happens when people have a genetic change in a gene called FOLR1 that prevents their brain from getting enough folate, a B vitamin it needs to work properly. This approval means adults and children with this specific genetic change can now use this medicine as a treatment.
WHY IT MATTERSFor the first time, patients with confirmed FOLR1 variants now have an FDA-approved treatment option specifically designed for cerebral folate deficiency, moving this condition from having no approved therapy to having a targeted treatment available.
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