14th International Congress of the WMS
September 9-12 2009, Geneva, Switzerland
 
The 14th International Congress of the “World Muscle Society WMS” was held from 9 to 12 September 2009 in Geneva, Switzerland. This meeting brought together professionals working in the field of neuromuscular diseases and aimed to establish multidisciplinary collaborations between international research teams, thus encouraging the development of scientific projects and therapeutic approaches.
 
Three main themes were developed for the program during these three days:

• new methods for assessing disease progression in neuromuscular disorders
• knowledge of the extracellular matrix in healthy and diseased muscle
• advances in the treatment of neuromuscular diseases

 
Two neuromuscular diseases were highlighted with the announcement of major advances in their field: Duchenne muscular dystrophy and spinal muscular atrophy (SMA).
 
Concerning Duchenne muscular dystrophy, Dr. Francesco MUTONI's team at The Dubowitz Neuromuscular Center at the ‘Institute of Child" in London (UK) reported the results of clinical trials using oligonucleotides (AVI-4658) that target exon 51 of the dystrophin gene. This trial, conducted on 7 patients who received AVI-4658 intramuscularly, shows dystrophin expression in the injected muscle of all treated patients at 3 and 4 weeks post-injection. These results are published this month in the ‘The Lancet Neurology’ (Volume 8, Issue 10, Pages 918-928, October 2009). At the same time, J. van Deutekom’s team (Prosensa Therapeutics BV, Leiden, Netherlands) announced the remarkable results of the Phase I/IIa clinical trial using the PRO051 compound and also targeting the exon 51 of the dystrophin gene. This is the first attempt ever made by injecting subcutaneously and therefore systemically, in Duchenne patients. Data analyzed on muscle biopsies from 12 patients, 43 and 78 days after treatment, showed an increase in dystrophin expression and some patients showed improved muscle function.
 
For SMA, Dr. Burghes (Ohio State University, Columbus, USA) and his team have obtained very promising results in SMA mice. These mice were injected intravenously at birth with an AAV-9 vector allowing the expression of the SMN protein. These mice usually do not survive more than 16 days after birth, while mice injected in this study are still alive today, more than 300 days after treatment. The objective now is to develop this approach in large size animal models.
 
Finally, in the poster sessions, the prize for the best presentation was awarded to Valerie Robin, a student in Luis Garcia’s group  (U974, UPMC, Institut of Myology, Hôpital de la Pitié Salpêtrière, Paris, France) for her poster describing a new therapeutic approach allowing to stabilize dystrophin in Becker muscular dystrophy.
 
The next WMS congress will be held in Kumamoto, Japan from October 12 to 16, 2010.
 
France Pietri-Rouxel
Researcher , CNRS
UMRS974 L. Garcia's team