Rare Disease and Orphan Drugs Journal (RDODJ) is an international, peer-reviewed, open-access journal dedicated to advancing research in rare diseases and orphan drugs. The journal publishes cutting-edge studies on the genetics and molecular mechanisms of rare diseases, diagnostics and research on undiagnosed conditions, epidemiology and database analyses, pathogenesis, precision medicine, gene and cell therapies, drug repurposing, and translational research from preclinical to clinical stages. RDODJ also addresses regulatory policies, health economics, healthcare organization, and social science aspects, including patient needs and quality of life. Committed to fostering collaboration and knowledge sharing among researchers, clinicians, and patient organizations, RDODJ is indexed in leading databases such as ESCI and Scopus and is set to receive its first impact factor in June 2025.