CureCN @ Rare Disease Day 2019
The 12th edition of the “Rare Disease Day” will take place on 28 February 2019. The main aim is to raise awareness for rare diseases and to improve access to treatment and medical representation for individuals with rare diseases. On and around this day, members of the rare disease community like patients and their families, policy makers, researchers in the field, as well as hundreds of patient organisations from all over the world will hold awareness-raising activities.
In the framework of this day, the French Crigler-Najjar Association (AFCN) participated in an event that was organised by the “Alliance Maladies Rares” at the University of Evry on February 21, 2019. Marylène Beinat (president of AFCN) together with Fanny Collaud (researcher at Genethon) presented the EU project CureCN in a mini-conference and informed about the Crigler-Najjar syndrome at an information stand. The audience, mainly students and representatives from other patient organisations were very much interested in a disease they had never heard of before. Marylène Beinat and Fanny Collaud were available to answer any questions regarding the CureCN project, the gene therapy, how to become a researcher, and the funding opportunities within the yet biggest EU Research and Innovation programme Horizon2020.
The audience realised that gene therapy poses a great challenge for the researchers as well as a great hope, not only for the Crigler-Najjar patients, but also for persons affected by other rare diseases that could be treated with the same technique.