Clinical Trial Landscape of Duchenne Muscular Dystrophy in Europe: A 10-Year Retrospective Analysis

Background and Objectives: Duchenne muscular dystrophy (DMD) is a rare and progressive genetic disease with limited treatment options. Clinical trials remain essential for the development of new therapies and may provide patients with access to new potential treatments. This study aims to describe the clinical trial landscape for DMD therapies in the European Union and European Economic Area (EU/EEA) between 2016 and 2026. Materials and Methods: A retrospective descriptive analysis was conducted using publicly available data from EudraCT and the Clinical Trials Information System (CTIS). Interventional clinical trials investigating medicinal products for DMD were included if they were active between January 2016 and June 2026 and had at least one investigational site in an EU/EEA country.

Results