Fabry Disease Insights

In their study, Chu et al. (2025) discuss the current treatments for Fabry disease, highlighting the use of enzyme replacement therapies (ERT) with agalsidase α and agalsidase β. Despite their effectiveness, these treatments face challenges such as high costs, short half-lives, and the potential for the body to produce neutralizing antibodies against them. The authors also explore the role of Migalastat, a chaperone therapy that enhances enzyme activity, noting its approval in many countries but also its limited application to patients with specific enzyme activity profiles.

Chu et al. (2025) further examine the development of new therapeutic drugs, including advanced ERT therapies, drugs that are resistant to neutralizing antibodies, and substrate reduction therapy drugs. They emphasize the continuous discovery of new pathogenic mechanisms and adjuvant therapeutic drugs, which contribute to the evolving landscape of Fabry disease treatment. The article serves as a summary of existing and potential drugs, aiming to assist in the selection of suitable and effective treatments for the disease.