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
It’s New Year’s Day 2015 and I think Janus, the two-headed Roman God who looks forward and backwards at the same time, is a fitting representation of my life right now. I need to tell you all that I have had to make a difficult decision this year—at the end of this month I will…Continue
We’ve been working toward this moment for years. And the time for action is now.
When I started with PPMD in 2009, Pat Furlong and I talked a lot about what it would take to end Duchenne. We knew it wouldn’t be one thing, and we knew it wouldn’t be easy. We believed that only a combination of therapies could address the entire disease within the body.…
As we finalize the agenda for this year’s Connect Conference, I am once again amazed at how much of a challenge it is to squeeze in all of the talks on different therapeutic approaches to treating Duchenne—in fact, there are too many to cover comprehensively this year. Sometimes it’s good to remember that mutation-specific approaches like exon skipping, although promising and…Continue