Ed.

Every human should at all times have equality in probability, not equality in outcome. NNT.

[Ed. @VerEduard]

@RNAiAnalyst Somewhere around Franken? Nürnberg perhaps?

[Ed. @VerEduard]

Quackary, Dr Anonymous Podcaster PrASSad, Crazy Beth Hog. Good riddance!

[steve usdin @steveusdin1]

There have been a lot of allegations that under Makary FDA leaders overruled staff – something Makary denied. Now there are receipts. Acting CDER Director Michael Davis placed a memo [https://t.co/JKcmDM4SsI] in the record outlining how his predecessor, Tracy Beth Høeg, repeatedly overruled career staff to quash approval of an sBLA from Sanofi for Tzield teplizumab to delay progression of type 1 diabetes.
Davis’s memo calls into question the integrity of the commissioner’s national priority voucher (CNPV) process, highlighting how it empowered political appointees to overrule career staff without providing evidence or documentation to support decisions. Davis also contradicts former Commissioner Marty Makary’s assertion that drugs were selected for CNPVs based on nominations from review staff.
Much more at: https://t.co/NZ4NrbX7kC

[Ed. @VerEduard]

@biggercapital

[Michael Bigger @biggercapital]

La Koka Racha La Koka Racha

[Lauren Holder @laurencurehd]

Here's the link for the STAT News podcast episode today: https://t.co/pjawyfzVPA

[Jeff Miller @JeffMillerTime]

https://t.co/SsyTOT8YIA

[Australischer Austauschstudent @VceOfReason]

Berichte über den von Rupert Lowe veröffentlichten Rape Gang Inquiry Report, der die Vergewaltigung von über 250.000 britischen Mädchen offenlegt:

ARD: 0

ZDF: 0

NDR: 0

RBB: 0

HR: 0

BR: 0

WDR: 0

SR: 0

SWR: 0

MDR: 0

Der Bericht ist tausendmal schlimmer als Gesänge auf Sylt.

Tausendmal schlimmer als ein Kaffeekränzchen in Potsdam.

Tausendmal schlimmer als der Fall George Floyd.

Und tausendmal schlimmer als die Beziehungsprobleme von D-Promis.

Unser objektiver Qualitätsjournalismus.

[Ed. @VerEduard]

@DesertDweller93 @Mitchell_A_C

[Alexander Stahel 🌻 @BurggrabenH]

Where are you going with this @piersmorgan? I never heard of the critic you mention below. But that is irrelevant.

Let's assume, for the sake of argument, that the final figure of 250,000 victims is overstated by 5%. Does that suddenly make the remaining 237,500 victims unimportant?

It's remarkable how, when faced with a catastrophe of this scale, some people's first concern is not the victims, the perpetrators, or the institutional failures—but whether they can shave a few percentage points off the total.

The inconvenient truth here is that the real number could just as easily be higher. We don't know, because the public was denied transparency for years. And the police literally refused to do its job.

Where I’m sitting, what you’re doing looks remarkably similar to what the police, politicians, and parts of the media did for years: divert attention from a deeply disturbing reality by obsessing over the margins instead of confronting the central issue.

You focus on disputing extrapolation, classifications, and technicalities, while hundreds of thousands of victims remain the uncomfortable fact at the heart of the story. And the rapes are ongoing - right now!

The deeper question here is whether aspects of political Islam are compatible with the values that underpin Western liberal democracies.

That is the debate people want to have—and the one too many institutions and people like yourself have spent years trying to avoid.

@RupertLowe10

[Peter Mantas @peter_mantas]

First off, this is an incredible day for HD patients and families. A generational, devastating, slow-bleed disease with no disease-modifying therapy now has the first ever GT in sight in the US. The rollercoaster has been real, the wait has been brutal, and today’s FDA re-alignment is long overdue. These families deserve this. I’m genuinely happy for every one of them.

From a capital markets perspective, $qure is incredibly undervalued at current levels.

The 3-year data that was always sufficient is now formally accepted as BLA-grade. No sham surgery — standard-of-care confirmatory control instead. RMAT added to Breakthrough and Fast Track. UK MHRA MAA filing Q3 2026, with the brand new MHRA-FDA liaison programme announced yesterday providing direct transatlantic alignment. Gulf states real. AMT-260 readout Friday now worth more from a platform validation standpoint. And Sung’s recent public comments about his patient cohort suggest the 4-year data will be compelling.

Sell side will take time to update models and they need to answer “what is the US worth, what is the UK worth, what does early label access look like.”

But at $81 with Prasad at CBER, no MHRA movement, and none of today’s catalysts, the market found fair value.

Today’s setup is materially stronger on every dimension. The models will catch up.

The compounder awakes.

$qure

[Houman David Hemmati, MD, PhD @houmanhemmati]

I totally agree with my friend @PeterDiamandis - it's DEEPLY unethical to deny or delay patients access to medicines that can save or dramatically improve their lives for bureaucratic or technical reasons.

Last week, I openly challenged former head of ophthalmology at FDA on this, frustrated that FDA has made it impossible to develop preventative treatments for intermediate macular degeneration (earlier form of AMD just BEFORE you are permanently blinded) by demanding treatments that dramatically improve vision before you've lost any vision.

Watch the video below. I argue that FDA's behavior has been akin to denying Stage 1 cancer patients a drug that works, forcing them to develop Stage 3 or 4 cancer so the clinical trial can demonstrate a larger potential benefit. This is backwards thinking.

The former FDA official defends his position by saying you can't create a preventative drug that doesn't work in 100% of people. WHAT? We give cholesterol and blood pressure medicines to tens of millions of people, many of whom will still get a heart attack or stroke (albeit often delayed), and many of whom will die of something else. Why take that CHOICE away from physicians and patients?

As long as I am alive, I am committed to bringing back COMMON SENSE & a patient-first approach to how drugs are regulated. The federal government is not the patient, nor it is the physician.

The FDA's role is clearly to be one important gatekeeper - to review clinical and other data to be sure there's a meaningful chance of efficacy outweighing safety risks and making those facts available to the public.

Once a drug is approved, there are THREE other layers of protection:

-Patients decide whether or not to take a drug
-Physicians decide whether or not to prescribe a drug
-Insurance companies decide whether or not to pay for a drug

I urge a move to this common sense approach that empowers the American PUBLIC to make informed decisions about their health as opposed to having very smart/committed & very well-meaning government officials limit our choices.

Our lives, our health, our vision, our ability to enjoy a high quality life depend on it.

Thank you @ArshadKhanani for moderating such an incredible panel & conference last weekend!

[Ed. @VerEduard]

Banger!

[Peter Mantas @peter_mantas]

This is getting silly. Adds:

$CLPT
$QURE
$MXCT
$RGEN

Remember, it’s all going to zero if the world ends because no one will consume drugs and the bios wont need to be processed anymore!

[Ed. @VerEduard]

@peter_mantas

[Lauren Holder @laurencurehd]

Today feels like a victory for the entire #HuntingtonsDisease disease community.

After months of uncertainty, difficult conversations, and tireless advocacy, we received the news that uniQure will be able to move forward as originally planned.

For many of us, like myself, this is deeply personal. We know what it means to watch the clock in HD. We know what it means to live with the reality that time lost cannot be recovered.
That's why this moment matters so much.

It's also important to acknowledge that this happened because people spoke up - patients, families, caregivers, researchers, clinicians, and advocates came together and REFUSED to let our voices go unheard.

We shared our stories. We signed petitions. We met with lawmakers and regulators. We educated others about what delays mean in a progressive, fatal disease where every day matters.

Today is a reminder that patient voices matter. Lived experience matters. Advocacy matters.

To everyone who signed, shared, spoke out, listened, supported, and stood beside this community: THANK YOU.

Let's take a moment to celebrate what we accomplished together.
Today, it feels like we were finally heard.
#TimeMatters #StandUpSpeakUp4HD #DelayIsDamage #RareDisease
@Help4HDI @FDA_KyleD @US_FDA @HDYOFeed @hdfcures @HDBuzzFeed @FDACBER @RepAuchincloss
@houmanhemmati
@JRenz0418
@Christina4HD
@rachelreising96
@BrittanyMegan10
@POTUS @senronjohnson @flsenrickscott

[Ed. @VerEduard]

@laurencurehd Bravo!!!!

[Adam Feuerstein ✡️ @adamfeuerstein]

Re $QURE news, I texted w/ Lauren Holder, a Huntington’s disease advocate who is also living with the early stages of the disease. Here's what she said:

“I'm literally crying right now. I'm so happy that I don't even know how to put what I'm feeling into words.”

She added, “This is the best-case scenario for our community,” she added. “This happened because of advocacy. This happened because dedicated patient advocates refused to give up, because this community continued to show up, speak up, and fight, even when it felt like no one was listening. Today, it feels like we were finally heard."

[Ed. @VerEduard]

Time to dust off this banger @MartinShkreli

[Martin Shkreli @MartinShkreli]

$QURE 5-10x from here

[Ed. @VerEduard]

Congratulations to the HD community! Hoping that this reach all of you quickly! #TIMEMATTERS #HUNTINGTONSDISEASE #AMT130 $QURE

[Peter Mantas @peter_mantas]

First off, this is an incredible day for HD patients and families. A generational, devastating, slow-bleed disease with no disease-modifying therapy now has the first ever GT in sight in the US. The rollercoaster has been real, the wait has been brutal, and today’s FDA re-alignment is long overdue. These families deserve this. I’m genuinely happy for every one of them.

From a capital markets perspective, $qure is incredibly undervalued at current levels.

The 3-year data that was always sufficient is now formally accepted as BLA-grade. No sham surgery — standard-of-care confirmatory control instead. RMAT added to Breakthrough and Fast Track. UK MHRA MAA filing Q3 2026, with the brand new MHRA-FDA liaison programme announced yesterday providing direct transatlantic alignment. Gulf states real. AMT-260 readout Friday now worth more from a platform validation standpoint. And Sung’s recent public comments about his patient cohort suggest the 4-year data will be compelling.

Sell side will take time to update models and they need to answer “what is the US worth, what is the UK worth, what does early label access look like.”

But at $81 with Prasad at CBER, no MHRA movement, and none of today’s catalysts, the market found fair value.

Today’s setup is materially stronger on every dimension. The models will catch up.

The compounder awakes.

$qure

[Help4HD @Help4HDI]

Breaking News: The FDA Approves the BLA Submission for AMT-130 through the FDA Accelerated Approval Pathway
A statement from our CEO, Katie Jackson: “Today marks a turning point in medical history for HD in the United States, as the approval of the BLA submission through the accelerated approval pathway for AMT-130 for Huntington’s disease transforms hope into tangible progress—proving that urgency and innovation can rewrite the future for patients and families. I have had the honor of working on projects with the FDA since 2014. The one thing I have learned over the years is that the partnership between the FDA, the trial sponsors, and the HD community is the only one that will help expedite drug development and get products over the finish line. I am so thankful to the clinical trial participants more than anyone else for making the brave decision to participate in a clinical trial, to the 51,000 people who signed the petitions, uniQure for fighting through, and the FDA that listened to us and utilized regulatory flexibility through one of their promising pathways to get this to the next step. We are truly grateful. Today is a historic day, and I am so thankful to uniQure and the FDA for allowing me to be a part of it. This milestone proves that when science and determination unite, we can bring light to even the darkest of battles and offer new possibilities for generations to come.”
https://t.co/72V9UfLPrc

[. @viatoCEO]

We just got a preview of a durable clinical benefit for $QURE AMT-130 at the 4yr mark and the market shrugged its shoulders.

The FDA will have no choice but to grant accelerated approval. $60+ share price incoming.