IMPROVING ACCESS TO DIAGNOSIS, TREAMENT AND CARE
    FOR PATIENTS WITH RARE DISEASES

    Patient organizations

    Industry


    Novartis Gene Therapies

    Healthcare professionals and research organizations

    Forschungszentrum für das Kind, Universitätskinderspital Zürich – Eleonorenstiftung

    Prof. Dr. med. Philipp Latzin

    Prof. Dr. Pascal Escher

    Prof. Andrea Superti-Furga, Service de Médecine Génétique – CHUV