making muscle wasting history

The recent AVI/MDEX annoncement is great news. Look at the press release on News on our home page. Biopsies for one young person showed over 50% for skipping exon 51. We would all hope that long term administration would show clinical benefit at this level. This is a great achievement and can pave they way for further trials to understand dosing and oligo design. The PMO appears to be safe even at high dose levels.

So what next? Action duchenne is working with AVI in our joint project to help move ahead with other exons and get enough data to bring these drugs to the market.

But WHEN Nick???

It really is hard to say as the next steps are in the hands of AVI. I can assure all our families and our supporters and donors that we are doing our best to move things along as fast as we can. Your continued support to AD and the DMD Registry is vital.

As soon as we have more news from the US I will keep you all posted. But this is a great result and vindicates our long term strategy of supporting this technology.