Anonymous23 days ago
On March 10, 2026, the FDA approved leucovorin (Wellcovorin) as the first treatment for cerebral folate transport deficiency caused by FOLR1 gene variants — an ultra-rare condition affecting roughly 1 in 1 million people — based solely on published case reports rather than clinical trials. The approval represents a significant retreat from the Trump administration's September 2025 announcement that suggested the drug could help hundreds of thousands of children with autism, a claim the FDA explicitly declined to support after finding insufficient evidence.