Lucerastat is primarily investigated for its potential therapeutic applications in treating rare genetic disorders, particularly those related to glycosphingolipid metabolism. It is being studied as a substrate reduction therapy agent for Fabry disease, a condition characterized by the accumulation of globotriaosylceramide (Gb3) due to enzyme deficiencies. By inhibiting an enzyme involved in glycosphingolipid synthesis, Lucerastat aims to reduce the buildup of harmful substrates in cells, potentially alleviating symptoms such as pain, kidney dysfunction, and cardiovascular complications. Its oral administration offers a convenient treatment option, and ongoing clinical trials are evaluating its efficacy and safety in improving patient outcomes.