What the project is about
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. MYOCURE explores strategies to overcome the key bottlenecks that hamper muscle-directed gene therapy.
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