FDA & Government News

FDA Approves Therapy for Cerebral Folate Deficiency

Decision addresses a rare neurologic disorder linked to folate transport impairment.

Share

The FDA has approved Wellcovorin (leucovorin calcium) tablets for treating cerebral folate deficiency in patients with confirmed FOLR1 variants, marking a first in treatment options for this rare genetic disorder. This condition severely impacts folate transport into the brain, often leading to developmental delays, movement disorders, and seizures. The approval, supported by systematic reviews and real-world evidence, highlights the drug's potential clinical benefits over the disease's natural progression. FDA officials emphasized the significance of this milestone for affected patients.

Original Source(s)

FDA Approves Therapy for Cerebral Folate Deficiency

Decision addresses a rare neurologic disorder linked to folate transport impairment.

  • by Kathryn Wighton

  • March 10, 2026

  • 2 min

Related Content

Blood Test Forecasts Alzheimer's Onset

Plasma p-tau217 “clocks” estimate timing of symptoms years before clinical diagnosis

  • March 9, 2026

  • 2 min
High-Intensity Focused Ultrasound Offers Immediate Relief for Essential Tremor

Essential tremor is a neurological disorder that causes involuntary, rhythmic shaking. Unlike the tremors associated with Parkinson's disease, ET is typically an “action tremor.”

  • by

  • March 5, 2026

  • 7 min
Retinal Clues to Alzheimer’s Pathology

Study detected Chlamydia pneumoniae and inflammasome-related proteins in retinal specimens

  • March 5, 2026

  • 2 min