A Japanese company has announced that it is starting a clinical trial for an exon 53 skipping morpholino. I don't have any more details beyond this notice, but would think it's highly unlikely that…Continue
French investigators have demonstrated that antisense oligos delivered with a gene therapy approach can improve muscle function and structure over the short term in dogs that lack dystrophin,…Continue
Hi Everyone-- We are in the process of planning for next year's Connect conference (already!) and I would really appreciate some feedback from some of you who attended the conference in Denver this…Continue
We are just putting the final touches on the agenda for both the Connect Conference and the first annual DMD Therapeutic Development conference. Both conferences will take place in parallel in…Continue
PPMD is thrilled to award two exploratory grants as part of our ongoing grant program. Peter Arthur of the University of Western Australia was awarded $50,000 to finish testing a molecule called procysteine in animal models in preparation for a human clinical trial. Procysteine is an antioxidant that has…Continue
When parents of children with Duchenne enroll their children in a clinical trial there is often a sense of participating in something bigger than themselves—of course you hope that your child may benefit from a new therapeutic approach, but you also know that all those days of missed school,…Continue
The World Muscle Society Meeting in Monterey, California saw reports on a number of topics of interest to the community. On Thursday poster sessions covered advances in the next generation of exon-skipping and updates on various clinical trials.
Next Generation Exon-Skipping…Continue