Abstract

Anderson-Fabry disease (or Fabry disease [FD]) is an X-linked lysosomal storage disorder that can lead to neuropathic pain, reduced physical activity, and other debilitating symptoms, besides major organ involvement, such as kidneys, heart, and central nervous system, with possible premature death. Despite specific treatments, such as enzyme replacement therapy or migalastat, adult patients may still experience clinically relevant symptoms and events. Supportive treatments are recommended, but their use is associated with disease-specific challenges. Unfortunately, few specific studies on supportive care in FD are available. Some supportive treatments, such as angiotensin-converting enzyme inhibitors and angiotensin receptor blockers, may contribute to slow disease progression. Recently, SGLT2 inhibitors have shown outstanding results in reducing morbidity and mortality in chronic kidney disease and heart failure; however, specific studies on FD are not yet available. A range of other supportive therapies may be useful in patients with FD to counteract additional mechanisms, besides substrate accumulation, in the pathogenetic course of the disease or simply to manage symptoms. In this review, we discuss the state-of-the-art of supportive care in FD, which requires a multidisciplinary approach that can significantly boost therapeutic potential and patient-centered care.