Prednisone is a corticosteroid medication that helps reduce inflammation in the body. In this trial, it is being studied as an additional treatment alongside standard therapies for Fabry disease. It works by suppressing the immune system and decreasing inflammation in the heart muscle.

Enzyme Replacement Therapy (ERT) is a standard treatment for Fabry disease that provides patients with a functioning version of the enzyme that their bodies are unable to produce naturally. This therapy helps break down certain fatty substances that would otherwise build up in the body’s cells.

Chaperone Therapy (CHT) is another standard treatment for Fabry disease that works by helping to stabilize the patient’s own defective enzymes. This therapy helps the body’s natural enzymes work more effectively to break down harmful substances that accumulate in Fabry disease.

Fabry Disease – A rare genetic disorder that affects the body’s ability to break down specific types of fatty substances. The condition results from mutations in the GLA gene, which leads to the buildup of certain fats, primarily globotriaosylceramide (Gb3), in cells throughout the body. This accumulation causes progressive damage to tissues and organs, particularly affecting the heart, kidneys, and nervous system. The disease typically begins in childhood or adolescence and gradually worsens over time. Symptoms can include burning pain in hands and feet, decreased ability to sweat, dark skin spots, and gastrointestinal problems. As the disease progresses, it can lead to complications in various organs, particularly affecting heart function and kidney performance.