Tag: Migalastat

Over 1,000 disease-causing genetic variants have been identified in the *GLA* gene, leading to the rare inherited disorder known as Fabry disease. A targeted, oral treatment for this condition is **migalastat**, which is used to treat adults with specific, responsive mutations.

Fabry disease, a rare genetic disorder, affects an estimated 1 in 40,000 males in the US. For certain adults with this condition, a key question is: **what does Galafold do?** This oral medication works by stabilizing a specific enzyme to help manage the disease.

Fabry disease affects up to 1 in 40,000 births, and for a specific subset of these patients, Migalastat, marketed as Galafold, offers a targeted therapeutic option. This oral medication functions as a pharmacological chaperone, addressing the root cause of the disorder in individuals with certain genetic mutations by stabilizing their own deficient enzyme.