Our aim remains to encourage the existence and recognition of myology by gathering on the same site, basic and clinical research, care and education. Since 2005, the Myology Institute Association’s mission is to facilitate the coordination of the site’s activities and prepare, in the medium term, the creation of a foundation dedicated to myology.

• Identification of new mutations in the DNM2dynamin gene associated with a particular form of centronuclear muscular dystrophy (M. Bitoun, P Guicheney’s team U582).
  
• Study on the properties of neuromuscular excitability in Steinert myotonic dystrophy through a non-invasive measurement of the neuromuscular excitability cycle providing detailed information on the functioning of ion channels (in particular, sodium and potassium channels) and the state of the membrane potential. It provides information to complement the data offered by techniques traditionally used in clinical neurophysiology (D. Boerio, D. Hantaï’s team U582).
  
• Progress on exon skipping by AAV U7. After demonstrating the importance of the small U7 gene that encodes an anti-sense RNA in a mouse model of Duchenne muscular dystrophy, its effectiveness will be evaluated in dogs, in collaboration with the of Yvan Torrente’s Italian team (L. Garcia’s teamUMRS 787).
• Development of European collaborations: the establishment of an NMD-Chip network, a European project whose aim is to accelerate the initiation of state-of-the-art therapeutics and thus clinical trials. This network project is focused on the development of molecular genetics as a necessary tool for therapies concerning certain neuromuscular diseases.

• Harmonization of clinical evaluations for clinical trials in progress at the Institute of Myology, particularly with all the neuromuscular diseases reference centres throughout France: 1st meeting dedicated to Pompe disease .
  
  
• Launch of new trials :
  • Initiation of the Phase IIb PTC 124 trial in France, conducted by PTC Therapeutics. The Myology Institute is the principal investigator of this trial in France and which is conducted in about thirty centres in Europe, the USA, Canada and Australia. It aims to assess whether the PTC 124 molecule, a neutral flavoured powder, administered orally three times daily, improves walking as well as muscle function and strength, and whether it is well tolerated by patients.
    
  • MYOREG trial : inclusion of three patients with inclusion myositis, an inflammatory myopathy characterized by a slowly progressive, asymmetric, motor deficit, in a study that aims to develop the first phase I/II therapeutic clinical trial, promoted by the Myology Institute (treatment of inclusion myositis by injection of polyclonal regulatory T lymphocytes).

The research tissue bank, formerly known as BTR, has changed its name to MYOBANK.