Now we know Farkas was the one at the FDA the Chris Garabedian resigned over. He's still holding a grudge. Stains the FDA corruption with his "Got Ya" review. Farkas, He should resign.
Now we know Farkas was the one at the FDA the Chris Garabedian resigned over. He's still holding a grudge. Stains the FDA corruption
What!!! , they had FDA guidance all along. Farkas pull the rug out from under this trial. He wanted kids to suffer for the sake of the trial.
Farkas had it out to get SRPT, he may have been the problem all along with CG. Farkas did not lay out data, he laid out an unreasonable attack.
So now what? New Placebo with biopsies trial. Extended trial until they to over way over 18? or whatever Farkas changes the new goals too? All over Again, biopsies from "same" muscle group. With three independent reviewers? Or whatever Farkas wants next? Reports on PT and steroid diaries? Same type or all over the place age groups like BMRN's failed trial.
Farkas option to change goal as he likes on Becker and other data? Farkas should just resign.
This was about Farkas and his "You don't know the whole story". Got you SRPT. Farkas presented data that he knew would screw over SRPT. He's getting his revenge. This was not about KIDS, just FDA petty officials making a point. Did you see any Positives from Farkus or Dunn? Nope..l.they were attacking not with analyzed data but with dirty got yours. Kindergartner actions from Farkas.
She is the one that will go to a Congressional board to explain her actions, not Farkas. He should be asked to resign.
So now all rare DX trials will require dead or disabled kids to be a success? Big extended placebo trials will require many someones to sacrifice their lifes. No natural history unless agreed on BEFORE the trial.
They are not going to get it from current EXON51, How many parents would want to risk placebo and a wheelchair for their KIDs? I would be out of the placebo in a flash and into some other trial.
It's can't be done now. Natural History has to be the only way. The FDA wants lots of biopsies they can't read. Big trials with doomed placebo kids and data they don't know how to read.
The FDA want bigs drama, kids in placebo falling down and into wheelchairs to make a BIG data point. Then they can point and say "well we have a great placebo trial".
How many kids do they want to sacrifice? 50? 100? 1500? 1-2 Billion $ more for limited new data?
The neurologist MD made the comment that if the trial would have gone on longer with placebo patients staying off the drug the differences would have been dramatic. Meaning all the placebo children would have been in wheelchairs. They now walk.
Woodcock is the one that will have to answer to Congress sooner or later on SRPT. Many will have signed the letter and comment on her lack of action.
Looking to data it seems it older and lacks breakdown of Beckers and or DMD variations. Odd studies without recent exon type difference being reported or studied. SRPT may have done one of the frist studies in this area looking for matched data..
Johns Hopkins Children's Center, study I read it does not matter how steroid are given they all fail to walk with whatever system they use about the same time..