Ataluren conference call

PPMD had a conference call with PTC, the company that is developing Ataluren, last week Thursday. They didn't really come out with any new amazing information, they were just re-stating what I had posted earlier about the low-dose seeming to work for the boys in the study. They DID say that they were working on/considering individual INDs for the boys in the study, meaning that they could have access to the medicine before it's approved. I guess an IND can apply to any investigational drug, but they don't always restrict it to study participants. Which in this case is frustrating, since Seth got diagnosed after the admission to the study was closed. Pat Furlong from PPMD said that this is what is needed for the "regulatory package" to get it approved by the FDA, and it is a "fine line" since many other diseases are caused by premature stops as well. But to that I say, well, THOSE diseases were not part of THIS study, so it's not such a fine line to only include boys with DMD with premature stops. Right? And while she said that all boys would have access once it's approved, and we're talking about a time frame of months, in a previous paragraph she says that the FDA MAY require another trial-which would mean more than months......it just seems unfair that Seth can't get it. I am trying to be hopeful and positive, but if some boys are getting the medicine, I WANT IT TOO!!! My logical mind understands the process, but my mother mind says screw the process. We know it's safe, and that it worked for most of the boys in the study, and that's all I need to know.

Apparently there is some conference in July and THEN we will know what the regulatory path forward will be-whether they will submit or have to do another trial. Which reminds me-somebody had posted that on of the study endpoints was a 12 meter or less decline in the 6 minute walk test, and the low dose group had a 13 meter decline (compared to a 48 meter decline for placebo/high dose groups). Seriously? They may not be able to submit because they missed the end point by one lousy meter?

Going along with all that, PPMD is working on some project to restructure the regulatory process for medicines for rare diseases, which will help this whole process in the future, so that will be good, but again, I need it now. I am so glad that there is research that is so advanced now, and I know it's for the greater good, but what about Seth and other boys like him? What about EACH boy? You know? I just wish there was more of a sense of urgency for them.