None of the data points are more important then benefit. Again the ADL's are beyond simple data points. Are these kid demonstrating benefit? Its almost magic what they are able to do with DMD and ETEP. All the parents say YES. To the FDA yes. They were invited to speak to the FDA. Compassion for their suffering was not enough to approve by the FDA but is is a consideration. FDA got hammered for not caring by the patients. Apparently FDA not in the Compassion business.
By the same hand added suffering is a data point as real Side effects are documented.
the news for Dispersion was side effect and failure but they want a pass. THings aren't bad enough for these Families that hospitalizations and kidney failure woun't help? Really! Do drugs have to taste bad to work? no...
Competitors pressure is holding back approval. RNA and Bio Martin intent is to bleed and slow SRPT. It's only business deal to them.
Drug allow DMD boys to walk. A drug miracle! Live years off a ventilator. A miracle drug. Activities of daily living (ADL's) off the chart, a miracle! They are not just doing a 6 min walk test. They have hope. Unless the FDA is only looking at something else like #$%$ data from BO Martin.
Was there ever reports of that drug causing any of this? no.. Their bluffing a FDA approval and SRPT to pay them off. Or bled them for a deal.
Bio Martin is using the same strategy RNA used. Bio Martin has a clause to bail out if they see it fail. Bio M are nothing more monkeys in suits. DMD a commodity.
Your forgetting side effects. MD and Lawyers won't. They could sell it to second and third world countries. Bio Martin if trying to derail ETEP and SRPT.
My request to Yahoo is not only Ignore but an outright elimination of posters and post from the message list that I see. We are seeing sick addictions to posting, like watching your dog lick it Ballix all day , day after day.
Just guessing if anyone bought this out it would be worth more that today price. More that RNA's value. Price Per share this low is just unrealistic.
We know now that they will decrease walking as doses Beckers. For off steroids comparison, a trail would have to be conducted. IE years of studies.
If the FDA doesn't except gene skipping Becker like results with SRPT (second place to Beckers) then no DMD gene skipping drugs will get approved.
No drug is going to be better that Beckers Natural History. Etep is expected to be a Becker like treatment. If you're expecting a cure you're not getting the purpose of SRPT drugs. They delay failure. Beckers are receiving mismade proteins all their lives. With ETEP just a fraction. If these boys extend walking 6-9 more years, at this rate, and delay of ventilator 15-20 years. Its a drug worth having.
I question the amount of dosing frequency. Is scaring happening between doses? Second question I have is even with loss in meters, how long before a wheelchair from natural history. So far a delay of three years. Could this delay a wheelchair 6-9 more years? No ventilator for 20 years? Would slow release oral or patch smooth peak and trough of effective work times.
Terrorism/ cold Wars 21-22 century, Computer, Bombs and biological . SRPT has one key chemistry to ready make a counter defence or offensive weapons. We just saw a first open computer war, its here. SRPT need to be part of a rapid reaction force. That is what Our Army should be working on, funded.
Tactics have changed. SInce they can't play the 10 only bad data card with a larger trial. Now its? A Dream Drug, as in "you've got be dreaming the drug is not a placebo. Very thin I think.
FDA is concerned that the methods used to measure dystrophin were not adequately robust to support an NDA submission. This from the FDA, after the RNA trail failure. Again RNA failed to impress the FDA. We eat shiezt while the FDA come up with a reason to or not to approve RNA.
Lets see, McDonald sold a bad natural study to the FDA. FDA confusing Natural History and RNA failure while reviewing SRPT. THats it in a nutshell.
Getting site IRB approval is a rabbit hole of delay and unknowns. We must wait is all. FDA is the blind spot, Not the drug or CEO.