Advancement of Treatments for Rare Diseases

The Advancement of Treatments for Rare Diseases Conference will take place on 16–17 June 2026 at the Cyprus Institute of Neurology and Genetics in Nicosia, Cyprus. The conference programme focuses on the development, evaluation, and delivery of therapies for rare diseases across Europe. Key topics include orphan drug regulation, drug discovery and repurposing, clinical trial readiness, patient involvement, European Reference Networks (ERNs), health technology assessment, data infrastructure, artificial intelligence in drug development, and access to innovative therapies. Sessions bring together policymakers, researchers, clinicians, patient organizations, regulatory experts, and representatives from the biotechnology and pharmaceutical sectors to discuss current initiatives and challenges in the rare disease ecosystem.