The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) held today the III National Conference: The Future of Advanced Therapies for Rare Diseases, with the aim of promoting dialogue and critical reflection among the different agents of the healthcare system in order to highlight the development of advanced therapies for rare diseases and access for patients in Spain. The conference was made possible thanks to the collaboration of: Amgen, CSL Behring, PTC Therapeutics, Ultragenyx Pharmaceutical and Vertex.

Among the main conclusions, the participants agreed on the importance of the therapeutic and social value of advanced therapies and the need to work together to improve the approach to rare diseases. Facing the great challenges, both at European and national or regional level, concerning diagnosis, research, development, financing and access to these treatments.