How long will it be before it is known whether molecular patches can be used as a treatment for DMD?
This research project is funded for three years. We recruited all children into this study in 2009, and the clinical part of the study was completed in early 2010. The MDEX Consortium and AVI BioPharma are in the process of analysing the data for submission to the UK regulatory bodies. Preliminary data from this study was presented as Late Breaking News at the International Congress on Neuromuscular Diseases, Naples, Italy 17-22 July 2010.In summary – AVI-4658 was well tolerated and no drug related severe or serious adverse events were recorded with either single doses or cumulative exposure. AVI-4658 induced dystrophin protein expression in a generally dose dependent manner in boys from cohort 3 onwards. A total of 7 patients had displayed a clear increase of dystrophin expression in the post treatment biopsy.
