Rare Disease Day takes place today and it is celebrated worldwide. The main purpose is to raise awareness about rare diseases and their impact on patients’ lives in order to motivate responsiveness of the public in general, but especially policy makers, public authorities, industry representatives, researchers and health professionals.
Asphalion is involved in many projects related to the research and development of therapies for rare diseases. In this line, we are very proud to have been awarded by the European Commission for the project MYOCURE under the H2020 topic PHC-14-2015 – New therapies for rare diseases. This European Commission funded initiative is aimed at developing an innovative gene therapy platform to cure rare hereditary muscle disorders. Eight organizations included universities, public institutions and private companies from five different countries work together on this project, which is estimated to have a direct impact on 20.000 people in EU suffering from MTM or GSD II. Moreover, MYOCURE will contribute to the IRDiRC objective of achieving 200 new therapies for rare diseases by year 2020.
Confirming Asphalion implication and commitment in this matter, our general manager in Regulatory Affairs, Lidia Cánovas, will participate in the conference about “R&D and marketing of drugs for rare diseases”. It will take place next 7th March 2017 in the “X Conferencia annual de las plataformas tecnológicas de investigación biomédica” event in Madrid. As an expert speaker, Lidia will provide an insight on the opportunities and challenges in the development of Orphan Drugs for the treatment of rare diseases.
