Preliminary results-not good :(

Here's a link to the press release: Ataluren

The primary endpoint for the study was a change in the six minute walk distance, and there was no change in the study participants. I know they were checking other things, like dystrophin in the muscle and doing bloodwork, too, but I would think that if there was some big improvement in dystrophin production, they would have said something. They are still analyzing efficacy in patient subgroups, but I don't expect that to be good news, either. As it is, they are calling it a failure, and have instructed families whose boys were in the study to stop taking the medication. PPMD is going to have a conference call with the company at noon and will post the information on their website that afternoon, but again, I'm not very hopeful.

I realize that this is how these things go-trials fail all the time, I'm sure. But we, along with a lot of other families, really had our hopes up with this. And like I've mentioned before, other treatments are in only preliminary stages right now, so they are years out. The trial has let me live in denial that it's not going to be as bad as we thought at first, but it really is...it feels almost as bad as when he was first diagnosed.

Anyway, I'll post if anything interesting comes out of the conference call.