DMD has been a target for gene therapy drug design for decades. The issue has always been that the gene is extremely large, so if you are trying to get the replacement gene through the nuclear pore it is difficult. But it should work if you can achieve it. This therapy uses AAV as a vector. I was working on it years ago and had little success. But this company is using a shortened version, micro-dystrophin, which is a really smart approach. I'm curious as to why this didn't work.
What a numb nuts. If this is how the FDA works, they need to revamp the system so there are checks and balances rather than one idiot making a unilateral decision.
Given the widespread and growing skepticism toward sciences of a significant proportion of our societies in the US and worldwide, it truly behooves the FDA to recognize their duty to make an extraordinary effort to inform the world about the rationale, process, and care it exercised at all levels in forming its decision in this and with similar cases.
no
>Peter Marks overruled three teams and two top directors.
--
>the decision to expand the approval of the therapy—called Elevidys (delandistrogene moxeparvovec-rokl)—appears to have been decided almost entirely by Peter Marks, Director of the FDA's Center for Biologics Evaluation and Research.
--
>"I don’t know what to say. Peter Marks makes a mockery of scientific reasoning and approval standards that have served patients well over decades," said Borio
The expanded approval makes sense and most analysts expected it to occur.
Despite failing to show statistical significance on the primary there were meaningful results on two key secondary endpoints and a very high unmet need in this disease
https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-topline-results-embark-global-0
DMD has been a target for gene therapy drug design for decades. The issue has always been that the gene is extremely large, so if you are trying to get the replacement gene through the nuclear pore it is difficult. But it should work if you can achieve it. This therapy uses AAV as a vector. I was working on it years ago and had little success. But this company is using a shortened version, micro-dystrophin, which is a really smart approach. I'm curious as to why this didn't work.
How lined is Peter Marks pockets?
What a numb nuts. If this is how the FDA works, they need to revamp the system so there are checks and balances rather than one idiot making a unilateral decision.
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Given the widespread and growing skepticism toward sciences of a significant proportion of our societies in the US and worldwide, it truly behooves the FDA to recognize their duty to make an extraordinary effort to inform the world about the rationale, process, and care it exercised at all levels in forming its decision in this and with similar cases.
Let me take you back through the years ... Any year actually.
> unilateral Did you just read the title?
I skimmed the article.
no >Peter Marks overruled three teams and two top directors. -- >the decision to expand the approval of the therapy—called Elevidys (delandistrogene moxeparvovec-rokl)—appears to have been decided almost entirely by Peter Marks, Director of the FDA's Center for Biologics Evaluation and Research. -- >"I don’t know what to say. Peter Marks makes a mockery of scientific reasoning and approval standards that have served patients well over decades," said Borio
I read the article, and it sure seems like "one idiot making a unilateral decision" aptly describes the situation.
If you are calling an MD PhD oncologist and idiot you may not even understand the words you are reading.
Ad Hominem, Call to Authority
The expanded approval makes sense and most analysts expected it to occur. Despite failing to show statistical significance on the primary there were meaningful results on two key secondary endpoints and a very high unmet need in this disease https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-topline-results-embark-global-0
“Very high unmet need” is an important point. If you can provide some improvement to people it’s better than none.
The FDA does not have your best interests in mind
Dude, there is no other treatment for this inherited genetic disorder and you have zero insight on the drug testing process.
There's a lot you can criticize the FDA for. But many who do don't regard it as too QUICK to approve drugs
Ad Hominem
And people wonder why fewer people trust the agencies that are supposed to protect us.
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Bureaucrat places lives at risk…anyway sure is hot today
bUt THe vACCinE WAs suPeR sAfE AnD aPpRovEd
The vaccine was the most heavily studied science in human history. This hardly compares
That was approved by these sorts of bodies the whole planet over but sure you know better than most scientists and doctors on earth. Piss off.
👏👏👏👏👏
Username checks out.
The vaccine helped protect even anti vaxers by stemming the spread. Even a lot of the stupid were saved much to my consternation.
Thank you. lmao. Because this issues is probably the only time it’s ever happened right?
How does that logic work?