5^th International Conference on Rare Diseases and Orphan Drugs

Orphan Drug Discovery: Orphan drug discovery focuses on identifying and developing medicines for conditions that affect relatively small patient populations and may receive limited attention during conventional drug development. Research in this area includes target identification, molecular screening, drug repurposing, preclinical testing, and the development of new therapeutic compounds. This session examines scientific innovations, development challenges, regulatory considerations, and approaches to making treatments more accessible to patients with rare diseases.