Testimonials

The aim of our project is to develop and study new cellular models of LAMA2-RD derived from patient stem cells (hiPSC). Thanks to this funding, the team has already derived hiPSCs from four patients carrying different mutations in the LAMA2 gene, and phenotypic analyses of the differentiated muscle cells are currently underway. These will enable us to identify the pathological mechanisms of LAMA2-RD in humans and new therapeutic targets.
For our small LAMA2 France association, our priority is to be able to provide practical support to researchers working directly on this muscular dystrophy. Project co-funding is an effective way for us to get involved, while enabling us to follow research projects in a concrete way, and thus improve the information we provide to patients and their families. It's a win-win partnership!