Research on rare diseases is critically important to the 300 million people worldwide affected by one of the approximately 8,000 known rare diseases. Studying these conditions has helped us better understand fundamental pathomechanisms, which may lead to the development of new therapies—not only for rare diseases, but also for more common disorders. This symposium focused on cutting-edge insights from basic and clinical science and their translation into the development of novel therapeutic strategies. The Research for Rare Networks, supported by the German Ministry of Research, Technology and Space, were proud to share innovative and exciting results and aimed to increase enthusiasm for rare disease research.

Rebecca Schüle (Heidelberg University Hospital) and Thomas Klopstock (LMU University Hospital, LMU Munich), on behalf of the Research for Rare consortium, cordially welcomed participants to the symposium, which took place from 22–24 April 2026 in the beautiful surroundings of Lake Starnberg. The conference was organised by the coordinating office of the German Research Networks on Rare Diseases.