ddums

[ddums @ddums81]

It’s becoming pretty obvious that the treatment of rare disease was the main reason Makary and Prasad were fired and that the FDA is determined to adjust course and fix the mess these two created. The poster child for the mess and unfair treatment of the rare disease community was AMT130. Extremely low probability that they don’t do an about face on AMT130 and grant AA. My odds are 90% chance.

[ddums @ddums81]

@peter_mantas Didn’t we already know this? Why is this news. Honest question

[The ₿itcoin Therapist @TheBTCTherapist]

You need to watch this interview with Jamie Dimon and understand how incredibly important the Clarity Act is for the banks to maintain absolute power over the people.

Jamie Dimon (aka the God King of fiat) speaks for the banks they are about to lose control to Bitcoin and crypto.

This is the single most important event in the institutionalization of crypto since the spot Bitcoin ETF was approved in January of 2024.

There are compromises being made on both the crypto lobbyist side as well as TradFi banking — Dimon does not want to compromise — he wants the banks to maintain absolute control like they have for decades — why wouldn’t he?

I am not a huge fan of Coinbase but Brian Armstrong is spearheading this campaign (with significant incentives) to offer a better opportunity for all investors in the U.S. and in crypto largely.

Dimon is trying to stifle innovation and Armstrong is pushing for it. Everyone should be rallying behind Coinbase and Brian right now — Dimon looks like a buffoon showing up on FOX to say he is “full of sh*t”.

This will eventually blow up in his face. The race to adopt crypto is much bigger than any one person, even Jamie Dimon. You cannot stop a technology whose time has come.

When the technology ultimately benefits the common person, those standing in the way of freedom technology will soon be run over.

[ddums @ddums81]

@BrianSkorney @RxRegA @peterbachmd Say one thing, do the exact opposite. That’s a sick and pathetic way to lead.

[ddums @ddums81]

@stuartbuck1 @Tim_Corr The problem is you shouldnt have Arnold Ventures people being the ones who decides what works. That sir is a conflict and politicizes the FDA. Not very hard.

[ddums @ddums81]

@mike98572986 @adamfeuerstein @statnews They need to show her the door. Get Arnold out!

[ddums @ddums81]

@mike98572986 It’s funny that there is a clear move by the FDA overlords to clean house of the Arnold Ventures rare disease decels, but CLPT and QURE trading way down. Market is wrong today.

[CP2 @LuckyPenguin10]

Each and every @US_FDA official with ties to Arnold Ventures needs to be removed.

These are the folks that enabled and supported Commissioner Makary's obstruction of promising rare disease therapies.

It's the only way to turn the ship and get U.S. biotech back on the right course.

That includes:

> Dr. Katherine Szarama — Acting CBER Director, former Arnold Ventures Director of Clinical Trials

> Dr. Tracy Beth Hoeg — Acting CDER Director, co-authored with Arnold Ventures-funded Prasad

And at the staff level, anyone else embedded through the same pipeline — including @AndrewNixonHHS.

Who am I missing?

Cc @SaraGonzalesTX @DC_Draino

—
@SenGillibrand | @lukaske
@SenRickScott | @ClareLattanze
@SenRonJohnson | @GraceCarnathan
@RepAuchincloss | @GeorginaBurros
@RickSantorum | @mattbeynon

—
$QURE $CLPT #FDA

[ddums @ddums81]

@mike98572986 @Dumbmuskrat Not soon enough

[ddums @ddums81]

@biggercapital Got reply then blocked by John Arnold yesterday. Badge of honor

[ddums @ddums81]

@johnarnold Ok I will play along. What if we do the opposite and raise taxes on gas, will that help? I’d like your response.

[ddums @ddums81]

@johnarnold Mini Soros, stop trying to push your rare disease worldview on the FDA.

[ddums @ddums81]

Agreed and you need to add rare disease to your list because that is where he isn’t just not doing enough, he is actually compromised by Arnold Ventures who is a leftist group run by an ex Enron trader turned Billionaire and he has successfully infected the FDA brass with his twisted world view. Look into it. It’s the big issue at FDA.

[ddums @ddums81]

@anish_koka And if you answer her, which is the only honest answer, then please just shut the fuck up.

[ddums @ddums81]

@anish_koka Even with her potential bias who should we trust more on AMT130, you or her? That is the question and the answer is obvious.

[ddums @ddums81]

@anish_koka If you say it’s you, you have the god complex just like Prasad and it’s so very obvious. Answer the question

[DesertDweller_ROAR_4_A_CURE_4_HD @DesertDweller93]

You got it wrong again. I was not comparing liver transplant to @uniQure_NV $QURE AMT130 only how tough a RCT would be in this population is and if you would risk your daughter's life in a similar situation.

I'll make it simple for you; would you have taken a risk of a 12-hour anesthesia and ventilator for a procedure for your daughter if you had a 50/50 chance of her getting nothing other than a death sentence and potential risk from the anesthesia itself?

Not to mention that during the 3 years of the sham portion of the trial where you received nothing other than 12 hours of anesthesia during the procedure, medical follow ups including invasive spinal taps, continuous loss of brain cells that will never come back, and the inability to take part in any other trial for a 100% fatal, hideous disease.

@DrMakaryFDA, who is a media whore, whenever he got the chance to talk, talked about real world evidence and how the FDA would use other measurements to determine efficacy for rare disease indications. There is no other database as comprehensive for a rare disease as the cUHDRS database and yet he wants patients to undergo a 12-hour sham procedure that carries immense risks.

I am not wrong on what the FDA is asking for. The original sham controlled portion had patients undergo 12+ hours of anesthesia, ventilator and drilling into the scalp. The drill holes did not go all the way in but they had drills in their head so they would think they got the treatment. This very likely contributed to a strong placebo effect for those patients when compared to the treated patients at ONLY 12 months when they were allowed to cross over. 4 of the 10 patients crossed over but the other 6 were no longer eligible for the treatment due to the progression of their disease and they no longer met the original screening criteria.

DON'T tell me I was wrong on the trial design. You are playing it down as if it is a "nick in the scalp" while downplaying the 12-HOURS OF ANESTHESIA and VENTILATOR.

You are being so disingenuous and you are ill informed. I post my profile so people understand who I am and I am fully transparent.

I have no issue with people questioning me or AMT130 results from an informed perspective, not one based on supporting some very unqualified professionals screwing the HD community at the FDA.

You say "What if 30% had blood clots..." Why ask a question for something that DID NOT HAPPEN during the trial as support for why the FDA should continue to require more patients to die needlessly in a future sham portion all in the name of science when they have data that allows a direct comparison for efficacy.

I have followed uniQure for over a decade and read and listened to every trial result over that timeframe. I've spoken to HD advocates who tell me of the patients that they know personally who have gotten the treatment. I've listened to experts IN THE FIELD, not some medical expert with almost zero knowledge of this particular debilitating disease and the trial. Sarah Tabrizi knows of at least 2 HD patients who received the high dose who were medically disabled who returned to work after treatment. She said she has NEVER SEEN this before in the HD community.

The prior FDA had not 1, not 2 but 3 Type B meetings and after ALL 3, agreed upon accelerated filing of a BLA in the first quarter of 2026. @VPrasadMDMPH decided he knew better and pulled that alignment only after officials from @johnarnold Arnold Ventures met with FDA officials to discuss "Items of Mutual Interest". What does a hedge fund manager have in common with FDA interests? Also, Makary lied when questioned about this on CNBC and said uniQure never had alignment which is a blatant lied disproven by the minutes of those meetings. Based on Prasad's public comments, even though he idiotically tried to hide behind "senior FDA official" only to give himself up, he blatantly lied about the sham procedure itself like you are doing by misrepresenting what it takes.

You continue to support these bought and paid for monsters for what purpose?

I make it clear, don't trust me, trust the experts in the field and you are quite clearly NOT ONE OF THEM. Sarah Tabrizi is and below are comments made by her describing the procedure on 3/30/26.

I realize after posting my transcript of Sarah that I didn't provide a link to her discussion so here it is if you want a chance of listening to an expert, not a podcaster and media whore.

https://t.co/Js17erSIv9

[ddums @ddums81]

Hey scientist Andy @AndrewNixonHHS any comment? Have you found your pink slip yet or are you still waiting?

[ddums @ddums81]

@anish_koka So you’re a cardiologist and you know better than the top HD doctors in the world? You have a ego and god complex just like Prasad and Markary which is probably why you love them. Stay in your lane.

[Biotech_hunter @biotech_hunter]

Why Marty Makary and Vinay Prasad decided to die on the replimune and uniqure hill is beyond me, just don’t move goal posts and stop asking for unethical controls, it’s bizarre $REPL $QURE