Precision medicine against myotonic dystrophy

Ruben Artero-Allepuz

Universidad de Valencia, Spain

Myotonic dystrophy is a hereditary neuromuscular disease that can manifest itself in any moment of life, both in childhood and in adulthood. It is characterized by a reduction in muscle mass, but also affects vision with cataracts, the cardiovascular system with heart rhythm disorders, the digestive system and the central nervous system.

Today there is no specific treatment for this multi-systemic disease that affects the entire organism, except for certain drugs that treat the symptoms and improve the quality of life of the patients.

Recently researchers have identified certain genetic alterations that hold promise for precision medicine. The project will develop strategies to compensate for the effect of the mutations that influence the progression of the disease with the aim of finding new treatments.

• Matthew Wood, University of Oxford, UK

• Javier Ramón Azcón, Institute for Bioengineering of Catalonia (IBEC), Spain

• Geneviève Gourdon, Institut IMAGINE, France

• Adolfo López de Munain, Institute Biodonostia, Spain

Therapeutic targeting of MBNL microRNAs as innovative treatments for myotonic dystrophy

€997,024