With over 95% of rare diseases still lacking approved treatments, there is a pressing need for specialized education in next-generation therapeutics. To address this gap, the Foundation for Rare Diseases (FFRD) is excited to announce the launch of their new Massive Open Online Course (MOOC) on FutureLearn: “From DNA to Cure: Gene and Gene-Modified Cell […]

We are pleased to announce the 2026 international “Dysmorphology Summer”, which will take place on August 20–22, 2026 in Palanga, Lithuania.  This annual meeting has deep traditions of bringing together clinical geneticists, medical geneticists, neonatologists, and other specialists working with rare diseases and syndromology. Participants review challenging diagnostic dilemmas, and explore the latest advances in diagnostic and treatment possibilities in an open and friendly […]

In December 2025, HLM RARE 2025 brought together EU policymakers, ERNs, patient organisations, researchers and industry leaders in Brussels to advance a stronger European ecosystem for rare and complex diseases. A key milestone was the launch of the Declaration on a European Innovation and Care Ecosystem for Rare and Complex Diseases, a political commitment structured […]