Advancing the development of new therapeutic products for patients living with rare diseases
The main impact of MYOCURE is to advance the development of new therapies for patients with rare muscle diseases. In particular, MYOCURE will impact directly on an estimated 20,000 people in the EU suffering from MTM or GSD II.
The MYOCURE project aims to exert a direct positive impact on the lives of MTM and GSD II patients by delivering novel treatment option(s) that will: (i) provide a long-term solution to life-threatening muscle disease and severe myopathy; (ii) exclude or significantly diminish the need for repeated sub-optimal medication by enzyme replacement therapy (ERT); (iii) be safer than the state-of the-art gene therapy options.
MYOCURE will have an impact beyond MTM and GSD II as the innovative platform technology will benefit most rare genetic diseases that affect muscle.
Enhancing quality of life and increasing competitiveness: Social and economic impact
The social and indirect economic impact of MYOCURE will be noticeable at different levels of society: (i) at the level of individual human being by improving the quality of life (QoL), health and survival; (ii) at the level of a family by improving the QoL of affected families through an improved independency of the patient from the family or relatives; (iii) at the level of society whereby increased QoL and autonomy of patients (and their families) will lead to an overall decrease in social costs associated with home or hospital care.
The technology platform developed within MYOCURE has implications beyond MTM and GSD II and would ultimately have a broader impact on patients and their families suffering from other rare muscle disorders. Hence, MYOCURE will foster improvement of medical knowledge and competitiveness of Europe in the field of gene therapy technologies which will further strengthen its indirect economic impact.
Contribution to IRDiRC objectives to provide 200 therapies for rare disease by year 2020
The outcome of MYOCURE will constitute the basis of a Phase I gene therapy clinical trial in MTM and GSD patients consistent with the IRDiRC objectives to provide 200 therapies for rare disease by year 2020. MYOCURE advances the development of a new therapeutic option for patients suffering from these rare inherited muscle diseases, as well as related preclinical research and animal model development.
Improving innovation capacity and integration of knowledge
MYOCURE will ultimately lead to a significant reduction in Europe’s healthcare costs associated with the treatment of inherited rare monogenic muscle diseases and in particular, MTM and GSD II. MYOCURE will enhance the EU’s expertise and strengthen the competitiveness and growth of companies by developing innovations meeting the needs of European and global markets in the field of orphan and rare diseases.