Re dosing of 9 boys in P2.
1) it was intentionally slow to start with to make sure it was safe. They dosed 1 boy, waited 6 weeks.. then dosed another 2, waited 4 weeks.. etc
They were also selective on who they dosed to get a broader range of boys as only dosing 9. Ie to get a good range across that 11yr old to 18yr olds.
2) with only 1000 boys in Oz with DMD. Only 500 non ambulant. Assuming 20% in Victoria thats 100. Assuming not all could make the journey to Melbourne clinic every 2 weeks and some who may have been on other trials as Melbourne clinic is used in global trials. That probably puts the eligible boys for that P2 trial at 60 ish. So its not like they had people jumping over themselves to be involved. NOW with the great P2 results out there,
P2b will be very different!!!P2b is 105 boys across 3 centres = 35 each centre combined with the excellent results I am assuming all boys will get first dose within 6 months of trial starting. Thats 1.4 boys per week per centre on average.
Also... this time last year people were b!tching about the options. Now we are complaining about how fast the dosing for P2b can happen!! A nice change..
Also, this time next year, my guess is the main focus of this forum will be the US trial.. hopefully open label. Lets see.
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