MYOCURE: Development of an innovative gene therapy platform to cure rare hereditary muscle disorders
The collaborative research project MYOCURE advances the development of new therapeutic products for patients suffering from rare inherited muscle diseases. The research specifically focuses on myotubular myopathy (MTM) and glycogen storage disorder (GSD) type II. MTM and GSD II are severe, potentially life-threatening rare muscle diseases for which there is no definitive cure and current treatment is suboptimal. These are attractive diseases for gene therapy since they are comprised of a diverse family of rare genetic diseases typically caused by single gene defects that often provoke significant morbidity and mortality.
MYOCURE explores strategies to overcome the key bottlenecks that hamper muscle-directed gene therapy. The MYOCURE project is funded by the Horizon2020 European Union programme for research and innovation with a total budget of about €6 million. Prof. Dr. Marinee Chuah from the Free University of Brussels (Vrije Universiteit Brussel) coordinates a multi-national consortium with long-standing experience and competences in the diverse fields required to fulfill the challenging objectives of the project.
Insights: Interview Series with MYOCURE Partners
Launched in 2000 the EU´s orphan designation program encourages research and development of...
The treatment of rare diseases remains a vast and largely unresolved issue. More than 7 000 such...
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