The congress Myology 2016, organised by AFM-Téléthon, is now coming to an end: over 900 international experts in the field (researchers, clinicians, doctors…) have compared their latest work for 5 days. This edition was undoubtedly marked by the beginning of the therapeutic era for neuromuscular diseases with the presentation of clinical trials results in human and the first lessons learnt from those trials.
Now again, we’ve witnessed presentations on the advances and the projects of gene and cell therapies for the heart, whether for cardiomyopathies or post-infarction heart failure. Therefore, P. Menasché (HEGP, Paris), leading the 1st French trial using embryonic stem cells for heart attack victims, spoke up. Drawing on his experience (4 patients now treated), he outlined the future development of this cell therapy approach, underlining the importance of trophic factors brought by the transplanted cells over the cells themselves. Lucie Carrier from Hamburg presented her work on mice for a gene therapy of genetic cardiomyopathy. For her part, Hélène Puccio (Illkirch) showed that gene therapy of cardiomyopathy in Friedreich’s ataxia works for mice, even in post-symptomatic cases. The vector used (AAV10-FXN) is being developed by the company Anapurna for a trial in human.
This 5th edition of the AFM-Téléthon congress was also the occasion for the young researchers financed by the Association to show their works to the international scientific community. Two of them received a prize for their oral presentation: Muriel Sébastien (Institute of neurosciences, Grenoble) and Pierre Klein (Institute of Myology, Paris).