Prof. Dollar

Keukenhof Tulip Gardens🌷 (Amsterdam, Netherlands)

The HD patients and families deserve AMT-130 Stop the politicization of FDA Fire FDA Commissioner Martin Mockery He has made a mockery of our healthcare system

For some visual context - here's a video of me and my dad dancing in 2017. The photos are 4 years later right before he passed away in January 2021. This is what progression of #huntingtonsdisease looks like. This is why a sham surgery is unethical in a #raredisease like HD. This is why we cannot wait. #TimeMatters and it's running out for us. @BeckyQuick @SenRonJohnson @SenRickScott @SenGillibrand @WhiteHouse @realDonaldTrump

$qure $clpt “All I can hope is that the combination of the patient voice, which is loud and clear, and the current data we have, and the emerging data will be heard,” stated Wild. “I hope the agency keeps an open mind and is receptive to what can and can’t be achieved, both ethically and practically.” That is all you need to know, Tabrizi, Wild and Sung keep screaming from the rooftops, if they had any concerns about the current and future data they aren’t going to continue to be out there speaking up for this treatment. Their eyes have told them enough that this cannot be placebo. All 3 of them have now seen many patients for almost 4 years, some 5. They know what a 4 year patient trajectory clearly looks like, they aren’t all exactly the same but they sure as hell know what they have never seen before. That is a hard thing to truly dispute. Stage 2 hd is no joke. @RepAuchincloss @SenRonJohnson @SenRickScott @SenGillibrand @WhiteHouse @FrankLuntz @SusieWiles @BeckyQuick @HDSA @HDBuzzFeed @Help4HDI @adamfeuerstein @MartinShkreli

“In the US alone, about a thousand people die every year from Huntington’s, and if that’s avoidable, it seems to me that there is a responsibility on every nation’s government and regulator to do what they can to help the development of these drugs.”

$qure $clpt Nice, more quotes from Ed Wild In a fireside chat at Advanced Therapies in London, Wild said he was concerned about the ethical implications of giving some participants a sham surgery despite the life-prolonging nature of AMT-130.  “The agency stated there would need to be a sham arm for the entire duration of the clinical trial and treatment arm,” Wild told BioXconomy. “Patients would have to go 3–5 years without treatment, which is a very long time, to be honest. A substantial portion may advance so much [in their disease progression] that they are no longer eligible for treatment.”  Wild admits that he and his colleagues were blindsided in the sudden U-turn the agency took after the recent US election, stating it vastly contradicted the agency’s previous position. “We were surprised that the agency took such a substantial step away from the advice it had previously given,” Wild elaborated. “It introduces an air of unpredictably that may be by design, but it makes life very difficult for patients.” “It’s very difficult to see how it becomes ethical to enroll people in a surgical trial, knowing that inevitably some would be too advanced to receive their treatment.” “All I can hope is that the combination of the patient voice, which is loud and clear, and the current data we have, and the emerging data will be heard,” stated Wild. “I hope the agency keeps an open mind and is receptive to what can and can’t be achieved, both ethically and practically.” “In the US alone, about a thousand people die every year from Huntington’s, and if that’s avoidable, it seems to me that there is a responsibility on every nation’s government and regulator to do what they can to help the development of these drugs.” @DesertDweller93 @Prof_Dollar @laurencurehd bioxconomy.com/modalities/fda…

Everyone following the @uniQure_NV $QURE AMT130 saga needs to read and retweet this. The rot in the @US_FDA begins at the top and needs to be fixed NOW! @SenRonJohnson @SenRickScott @RepAuchincloss @adamfeuerstein @l_e_whyte @laurencurehd @BeckyQuick turn up the heat.

Few understand this. $QURE is not some science project. They are one of the few biotech firms to develop a gene therapy and bring to commercialization w/partner. The delivery platform works. @RepAuchincloss @SenRonJohnson @SenRickScott @SenGillibrand @FrankLuntz @BeckyQuick

❤if you agree: Today, @US_FDA shouldn’t be trailing at all. If EMA says yes, it means science is sound, review is rigorous, and patients come first. @uniQure_NV: take this where it’s welcomed: EMA, UK, Asia. Let results speak. #Huntingtons #RareDiseases $QURE $CLPT $RGNX

$QURE Keep it up

all because this douchebag @johnarnold doesn't think rare disease patients' lives are worth anything easy to say until it is your family member who has DMD, SMA, CF, HD, etc.

This guy needs to go. Hopefully, he is next to be axed after Vinay

A full blown scandal. Ideologues funded by and aligned with an ex-Enron trader have been put in charge of the #FDA without any knowledge of how drug innovation works and see cost first instead of progress.

I don’t think people fully appreciate what centene approving use of amt-130 means even without FDA approval. An insurance company is willing to cover a $2m therapy because it fucking works $qure

Everyone following the @uniQure_NV $QURE AMT130 saga needs to read and retweet this. The rot in the @US_FDA begins at the top and needs to be fixed NOW! @SenRonJohnson @SenRickScott @RepAuchincloss @adamfeuerstein @l_e_whyte @laurencurehd @BeckyQuick turn up the heat.

$qure $clpt $rgnx “At least 4 overrules” where historically this doesn’t happen.

While we’re all watching the FDA, 2 papers quietly published 2 days ago flew under the radar for $QURE. botn.bio/p/the-paper-no…

Hundreds of individualized treatments for rare diseases could become available over the next decade — but only if government regulators handle the new therapeutics properly. At least that’s how Robert… trib.al/KMeCT0Q

👇the moneyed interest behind Prasad, @DrMakaryFDA and their hires at #FDA.

in July, Arnold Ventures’ chief executive, Kelli Rhee, met FDA commissioner Marty Makary for an introduction. A spokeswoman for the foundation said it played no role in getting people hired at the FDA. Covered extensively on $qure in the FT Dec article. ft.com/content/74b9b5…