IMPROVING ACCESS TO DIAGNOSIS, TREAMENT AND CARE FOR PATIENTS WITH RARE DISEASES Patient organizations Industry Novartis Gene Therapies Healthcare professionals and research organizationsForschungszentrum für das Kind, Universitätskinderspital Zürich – Eleonorenstiftung Prof. Dr. med. Philipp Latzin Prof. Dr. Pascal EscherProf. Andrea Superti-Furga, Service de Médecine Génétique – CHUV