Morris. you lab rats (said with affection) couldn't diagnosis a "zit" if it burst in your face. You and Pastuer wanna be, and Regulatoryfake are nothing but hacks who think that all that matters is your lab anaylsis and opinions. I have encountered your types often. You guys are the ones at the lunch table eating by yourself. You can get on a message board and sling insults at other posters because in your mind you think you are superior. Your not.
Try being a clinician, or a least co-vist a patient or 5 that live with DMD and have a heart to heart with the parents. Maybe that would motivate you to be a little less caustic. I wrote earlier that clinicians evaluate the entire patient not just their dystrophin level or the 6MWT. In fact, just stabilization would make me giddy. Not one of you have explained the lack of derioration of the boys and yes the two who stopped ambulating within weeks of the trial starting. Not one!!! Why. Steroids, now really!!! Brand new defintion of DMD...hardly. Because you have no sense of the clinical aspect of medicine. I do!! Oh, and FYI I trained at one of the most prestigious pediatric hospitals in the world where the lab rats and the clinicians collorborated all of the time. My, what progress was made at that facility in once before fatal childhood disease. See the focus was the child not precious egos. So go back to your rats and leave the clinical aspect of medicine to us unless you want to sit down and have a heart to heart with us clinicians and actually get to know the patients you think you are helping.
folks, you can hate all day long on other posters who're disagreeing with you and believe they are sent from hell to mess with you, it will not help your cause and it just creates distraction from the actual issue at hand
I’m really amused by your post. The FDA looked at the 6MWT data and determined the difference between the two groups could be explained by natural variation. Oh sure we could go by the testimony of the parents and using our hearts to determine if the therapy was efficacious. Or we could let trial sponsors have multiple endpoints to explore possible measures that might met the statistic rigors of clinical testing. You know, give them many apples and as many bites as they want. The problem with that is there would be ineffective drugs marketed because of a chance; more bites means more chances at find a statistical anomaly.
It appears you don’t know your field very well. I work with clinicians but it’s pretty far down the road. In my field, first we isolate the virus, then we do basic research, we then study vaccine candidates, express a viral sub-unit protein, highly purify it, bottle it, and then finally involve a clinician. After the trial the clinicians and the research scientists are co-authors on manuscripts. Research scientist aren’t going to back seat to clinicians. You should put your Johnson back in your pants and back off.
Your and the FDA's problem is you / they would rather see kids needlessly suffer and die while waiting several years for a safe drug to prove out in trials-- because for some bizarre reason you are all MORE CONCERNED with how it might look if the FDA had to pull a drug in the future, if it really proved not to work on the patient population after early approval than weather or not kids are spared suffering if it indeed does work as thought.
There is a reason why accelerated approval law was passed.
It goes way beyond the FDA ever having to backtrack to pull a safe drug that didn't end up being effective. That's not the primary concern. It's not about that.
Morris the first thing I would do is a meet some boys and parents with DMD and try looking at the disease from their standpoint. Then lets talk. It's amazing what brilliant minds and some people with just plain good ideas can come up with. The first rule I was always taught and constantly reminded of was listen to the mother. In these days I admonish my residents to listen to mother and the father!!!