As things stand, this novel compound is safe in rodents and non-human primates, and according to mouse models of the disease it allows dystrophin rescue and functional improvement in skeletal muscles, heart and brain following few weeks of treatment. Nonetheless, in the shorter term, we are supporting top quality medical studies on the management of the cardiac complications through the retrospective analysis of enriched databases “genotype/treatment/phenotype” to evaluate the benefit, if there is, of certain combinations of drugs prescribed to patients over the last decade, as well as we contribute on demand to ongoing clinical trials to accelerate the implementation of the best care practices.
We are conscious that there is still much to be done and that our initiative needs, more than ever, help and support.
President - Association Monégasque contre les Myopathies
Founder / Organizer - Only Watch