Brain Health

FDA Approves First Treatments for Rare Neurological Conditions

The FDA recently approved two groundbreaking treatments for rare neurological conditions. Avlayah (tividenofusp alfa-eknm) becomes the first drug approved specifically for neurologic manifestations of Hunter syndrome, a genetic disorder affecting brain function. Wellcovorin (leucovorin calcium) tablets received expanded approval for cerebral folate deficiency in patients with folate receptor 1 gene variants, marking the first treatment for this condition affecting brain folate transport. These approvals represent significant advances for patients with previously untreatable neurological complications.