Tim

@US_FDA fine, i'll do it

@BillBrewsterTBB @Macro_First @US_FDA @MartinShkreli

@Macro_First @US_FDA It’s a simulation. We need @MartinShkreli like my guy @Tim_Corr says.

Are you ready to shape the future of public health at the highest levels? The FDA is seeking an exceptional executive leader to serve as the Center Director for CBER—a role at the forefront of regulating life-saving biological products, vaccines, and emerging therapies. This is an opportunity to protect and advance public health on a national scale. You'll work on cutting-edge science, shape critical regulatory policy, and lead a team dedicated to ensuring the safety and effectiveness of biological products that save lives. Apply by April 3, 2026. #FDAJobs usajobs.gov/job/862056100?…

@US_FDA @MartinShkreli this is all you. $qure

Rare disease advocacy tip that nobody tells you: Don't argue emotion vs. the FDA. Argue their own words back at them. Every designation they granted, every pathway they agreed to, every meeting minute they signed is a receipt. The FDA generates its own paper trail. Your job is to make sure people see it. #RareDisease #PatientAdvocacy

$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…

worth reading. nice work on amt-130's story.

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.

$qure $clpt $rgnx @RepAuchincloss @SenRonJohnson @SenRickScott @SenGillibrand @WhiteHouse @FrankLuntz @SusieWiles @BeckyQuick @HDSA @HDBuzzFeed @Help4HDI

Looks like @Temple_West at the Financial Times was way ahead of everyone on this. His Dec 2025 reporting on Arnold Ventures' influence at the FDA independently confirms the personnel pipeline: Prasad + Szarama + Hoeg Ironically, John Arnold's most consequential trade may have happened long after his hedge fund career. H/T @ripper1399 $QURE $CLPT

If you're just hearing about Huntington's disease for the first time because of the AMT-130 story, here's what families live with every day: A parent watching their child lose the ability to walk, speak, and think. Knowing their other children might carry the same gene. Knowing there's no treatment. Watching it happen twice, sometimes three times, in the same family. That's who the FDA called "moneyed interests." That's who an anonymous official dismissed while calling the only promising therapy "snake oil." These families aren't investors. They're parents doing math on how many years their kids have left. Follow @Help4HDI. The fight continues. #HuntingtonsDisease #AMT130 #RareDisease

Ok, this is all snapping into focus. And candidly, the picture that's emerging is far more interconnected than anyone assumed re: what's happening to $QURE AMT-130 and the broader rare disease ecosystem. This also seems to explain Vinay Prasad's abrupt (second) resignation. If all this adds up — there's a specific law within the Standards of Ethical Conduct that becomes very relevant: 5 CFR § 2635.502, "The Impartiality Rule." To be clear, this is not an accusation — simply following the paper trail. Everything here is drawn from public records, sources at the bottom. Ok, let's start...

@AppleHelix assets.arnoldventures.org/uploads/Arnold…

Claude was able to find Vinay Prasad's CV from 2023 (for tenure application?). onlinelibrary.wiley.com/pb-assets/CV%2… VP actually received 2 separate grants from the Arnold Foundation. One from 2017-20, the other 20-23. And probably is still supported by the foundation. Total from 2017-23 was ~$4m. Most recently, the foundation was funding VP $800K per year.

$qure $clpt Huge story here, be sure to let your people know, this is the true story. Make sure Congress and Uniqure knows of this. This is the big investigation that needs to happen immediately. Be sure to retweet the heck out of this. This is a huge scandal and ethically a complete disaster for the FDA. Makary is definitely done after this one. This right here needs to go viral. @RepAuchincloss @SenRonJohnson @SenRickScott @SenGillibrand @WhiteHouse @FrankLuntz @SusieWiles @BeckyQuick @HDSA @HDBuzzFeed @Help4HDI @adamfeuerstein @MartinShkreli @laurencurehd @DesertDweller93 @Prof_Dollar @uniQure_NV

Funny little fact pattern. 1.Szarama wrote the Arnold Ventures letter arguing against external controls in December 2023; 2.She was appointed CBER Deputy Director October 8, 2025; 3.FDA rug pulled on AMT-130 22 days later; 4.No public recusal record exists. @adamfeuerstein @LizzyLaw_ @SenRonJohnson @SenBillCassidy @RepJasonSmith @RepAuchincloss @SenateAging

@docrodwong assets.arnoldventures.org/uploads/Arnold…

this is the first major mention i've seen of arnold venture's influence on this fda. arnold is said to be have been a major backer of prasad's research (i don't have proof of this, have just been told many times), and as wsj notes, szarama worked at arnold before prasad brought her in. most aren't aware arnold ventures was the major backer of ICER, the group that defines the value of medicines for the UK (which is set at a cap of roughly 30k pounds per human life year). it is also the reason the UK has dropped below basically all other developed countries when it comes to access to rare disease medicines. in short, arnold doesn't believe rare disease medicines are worth it, and it's natural to conclude prasad and others were bringing that philosophy to fda. now it's fine to have that view as a society (as the UK chooses to), but the trump administration represented the opposite, which is to accelerate access to rare disease medicines. wsj.com/opinion/one-ex…

@docrodwong onlinelibrary.wiley.com/pb-assets/CV%2…

@Tim_Corr thank u for the receipts. if anyone else has more would love to see.

Get this viral #IAmtheSwamp

This is me & my dad. My dad before HD changed him into someone I didn’t know. He suffered for years as HD slowly deteriorated his brain and then he chose to end his life on his own terms. An HHS official just called us “the swamp”. Make it viral #IAmtheSwamp @AndrewNixonHHS

This is how an @HHSGov / @US_FDA official speaks in public. Imagine the conversations and behaviors internally. Meanwhile, as they stonewall a promising therapy in AMT-130, Christina's father continues down the painful path of Huntington's disease. I suppose Christina's family must deserve it – because they're part of the "swamp." The "swamp" Nixon is referring to includes the ~48,000 individuals from the Huntington's community behind the two petitions delivered to the FDA. Petitions the FDA did not even review. @SenGillibrand | @lukaske @SenRickScott | @ClareLattanze @SenRonJohnson | @GraceCarnathan @RepAuchincloss | @GeorginaBurros It doesn't have to be this way. You have thousands of bipartisan constituents watching proudly of the actions you've taken so far. The latest being the press conference on Mar 10th that @SenRonJohnson held. For the sake of Christina and the millions of rare disease patients, keep going.