Ann: Trial Met Primary & Exceeded Secondary Endpoint Expectations, page-159

You made my day with that reply Mr. Learner - happy to support it 100% - and thank you.

Regarding the boys, I have been trying to make sure I fully understand where they are currently placed, and once again find my knowledge of the terms used in this industry is woefully inept. The info provided in the webinar showed that the suspension of deterioration/improvement in the condition of their muscles had reverted back to their starting condition at the check done at week 28, so they had in effect gained an additional 6 months of life ? Other than that, I am pretty sure I came across some assurances that any or all of the nine boys who completed the RCH trial and wanted to continue were being taken care of (or words to that effect) noted by Sam in a post way back maybe January but I now cannot find it - and I think he also and maybe Itsa recently made reference to them being included in the EMA 2B trial under an open something structure which I don't know the meaning of. So I would greatly appreciate anything you (or Sam or anyone else) can provide about that situation. From what I have read, an EMA 2B trial could be conducted as a series of trials in separate locations presumably in order to achieve the total number of participants the EMA will require. If this is the case, the task of maintaining the quality standards achieved here in Melbourne will be much more difficult won't it ? e.g. if it is divided between several countries speaking different languages ? Do the key company staff from Melbourne go over there for the trial to work with Dr. Price ? and if it means that our boys from the local trial are able to continue participating by means of another trial here in Melbourne, whatever result is achieved will by definition be made by the TGA and only apply in Australia, with other overseas authorities able to agree to them if they wish to ? And while Dr. Price will look after Europe from the U.S., what will Mr. Goolsbees role be ?

There are other aspects I would like to raise, but as a last one for now, I think it was Uboy who has asked in relation to an accelerated pathway "DoesTGA success depend on FDA /EMA approval ?"
My take on that is unequivally NO ! but again the TGA website strikes me as being pretty daunting in terms of its reporting etc requirements and could contain anything.
Nevertheless, I understand it was the company that, on the recommendation of their U.S. agent sought the views of the FDA as to what dosage they would accept for the DMD A trial in order to qualify for FDA agreement to a decisive follow-up B trial. Moreover, it was later pointed out by other posters that the company had been informed by the 3 large shareholders group from whom they were seeking a capital placement in order to fund the trial that they would agree to it on the condition that the go ahead to proceed with the trial had been obtained. So, having been informed of the FDA's position the company then arranged the RCH trial based on a 25gm/weekly dose, and quickly announced the capital raising and trial arrangements. All of which seems very orderly. But the question I have is why didn't the company seek the TGA's view as to whether they would agree to the trial being undertaken with a variable dosage range based on their experience with the MSG trial they had already conducted, and with open pathway options included, rather than stipulating the 25gm/weekly dosage ? I feel that it is the TGA's responsibility to decide whether things such as blinds, double blinds, placebos etc. are required, and no-one elses. They have not provided us with a clear explanation of these events, or the reason why they chose to conduct the trial at home here in Australia. And then of course, the company sent Dr. Woodcock and others to report on the results of the trial seemingly on the first plane out, with no mention of having first similarly informed the TGA , which I would have thought would be the first priority.
There are still other aspects I wish to clarify but will stop here for now.
As a last general comment I think the current events in the U.S. are placing the entire world recovery in a much more dangerous situation than it appeared to be just a few days ago, while on the other hand I have also come across a quite positive view that the EC, led by Germany's Mrs. Merkel and France's Macron are putting together an EC tax plan that may finally give the bloc the working structure that it has lacked, which if it is correct will probably be very helpful to our objectives over there.

And, with all those accents in use in the U.S. I think it someone different would be needed to coax investments from those good ole boys who still seem to have the family fortunes accrued during the first industrial revolution -
I think Dolly Parton still has all the smarts and charm to make a very effective effort over there ?