Post–marketing clinical study of leuprorelin acetate for spinal and bulbar muscular atrophy

Abstract

In Japan, many new drugs for neurodegenerative diseases have been developed in recent years, with translational research playing a key role, exemplified by leuprorelin acetate for SBMA. Clinical trials often fail to confirm the long–term efficacy of disease–modifying therapies for slowly progressive neuromuscular diseases, highlighting the need for post–marketing clinical studies. Real–world data, exceptionally patient registries, has become increasingly important when clinical trial data alone is insufficient. Our study evaluated the real–world efficacy of leuprorelin acetate for SBMA using trend–shift analysis, demonstrating a practical method for assessing drug effectiveness in post–marketing research.