Migalastat hydrochloride is primarily used in the treatment of Fabry disease, a rare genetic disorder caused by the deficiency of the enzyme alpha-galactosidase A. This condition leads to the accumulation of specific lipids in the body’s cells, causing symptoms like pain, kidney dysfunction, heart issues, and stroke. Migalastat hydrochloride works as a pharmacological chaperone, helping to stabilize and enhance the function of the defective enzyme. By doing so, it facilitates the breakdown of accumulated lipids, alleviating symptoms and slowing disease progression. It is particularly effective in patients with specific genetic mutations that respond to this mechanism of action. The oral administration of migalastat hydrochloride offers a convenient treatment option compared to traditional enzyme replacement therapies, improving patient compliance and quality of life.