Fahr’s Disease (PFBC)

Targeting Progressive Calcification in the Brain

Fahr’s Disease, also known as Primary Familial Brain Calcification (PFBC), is a rare inherited condition characterized by progressive calcium deposits in the basal ganglia and cortex of the brain.

Patients develop a range of debilitating symptoms, including movement disorders (like parkinsonism and dystonia), cognitive decline, psychiatric symptoms, and seizures, often beginning in mid-adulthood.

Etidronate, the active molecule in ZY-001, has shown promise in preliminary reports for reducing calcification volume and improving neurological symptoms in patients with brain calcification.

An ongoing European clinical study (CALCIFADE Trial, Leiden University, 2024-) provides international support for the concept that bisphosphonates may slow or reverse disease progression, collaborating Zy Therapeutics’ platform approach to brain calcification disorders. The CALCIFADE trial recruitment may be completed in 2026 with results the following year.