Wow! Interesting. :) Low dose "working" better than high? Have they looked at that dystrophin expression already?
http://www.parentprojectmd.org/site/DocServer/2010-04-16_Final_Summ...
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If we only look at the statistics results with RANCOVA method, can we say the low-dose Ataluren might be working for the boys with non-sense mutation? I am still very confused about efficacy results part of the article. Can you explain it a little bit more without math statistics terms?Thank you very much!
Melanie.
I do not know who made those summaries, PTC or Dr. Wong.
Either way, the results using RANCOVA (which was specified in the protocol and in my opinion the good approach to analyzing this data) satisfy 85% significance level (this means that the finding has a 85% chance of being true.)...usually one wants to see a 95% level.
Just based on the results presented yesterday, it is too early to say that the low dose works, more data analysis needs to be done, it might work for a subgroup. It would be good to put the kids on low dose and collect more data while completing the analysis including biopsy data BUT that might not be doable due to protocol (regulatory agencies might not allow this).
What we can say with certitude at this point is that the high dose does not work. That in itself needs to be explained before they move forward.
Melanie Sunny said:If we only look at the statistics results with RANCOVA method, can we say the low-dose Ataluren might be working for the boys with non-sense mutation? I am still very confused about efficacy results part of the article. Can you explain it a little bit more without math statistics terms?Thank you very much!
Melanie.
Ofelia
How are you certain that high dose does not work for any of the boys?
Brenda Wong's report states that Ataluren may have a bell-shaped dose-response curve so this means that the high dose could be effective for some. Many of the best responders to Ataluren may have been randomized into the placebo group so they will not show in the stats. As you probably know, the effectiveness of Ataluren depends on factors such as the gene sequence and then the location of a pyrimidine etc. I'm pretty sure that the high dose will work for some of the boys as there have been reports of it doing so from the 2a trial.
Is there some other information regarding the trial that I have missed which categorically says the high dose does not work for any?
Kenny
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