Prof. Dollar retweeted
CNPV for AMT-130. Every single day untreated for Huntingtons patients brings harm they can never reverse. The 4 month difference between AA and CNPV evaluation could be the difference for hundreds between being able to recieve lifesaving treatment or being too far gone to help.
Prof. Dollar retweeted
Sorry to add to the bad news today…
Novartis $NVS cites cases of previously unknown tumors possibly tied to its SMA gene therapy statnews.com/2026/11/05/nova… via @Jasonmmast @pharmalot
Prof. Dollar retweeted
$RARE is an example of recent gene therapy approval from August 2026 without the need for a real placebo-controlled trial and is instead using data from commercially treated patients. @FDA_KyleD please change the alignment with @uniQure_NV $QURE AMT130 to a similar design.
It will be unethical to follow HD patients for 3 years who never receive treatment until after the 3 years.
@laurencurehd
@Christina4HD
@JRenz0418
@rachelreising96
@geneveritas
@SenRonJohnson
@SenRickScott
@RepAuchincloss
@houmanhemmati
@adamfeuerstein
@armstrongdrew
@l_e_whyte
@temple_west
@ScottGottliebMD
@BeckyQuick
@davidfaber
@AllysiaFinley
@bradloncar
@docrodwong
@LuckyPenguin10
@POTUS
@SenateAging
@SenBillCassidy
@SenRandPaul
@WhiteHouse
@SusieWiles
@FDACBER
@FDA_KyleD
@DrOzCMS
@HHSGov
#Duchenne
#PrimaryImmunodeficiency
#RareDisease
#HuntingtonsDisease
#FDA #PatientAdvocacy
#PatientVoice
#TimeMatters
#RareDiseaseResearch
#RareDiseaseFramework
Prof. Dollar retweeted
$QURE We are now in the commercial launch chapter of the company. Time to start modelling what amt-130 is worth as a franchise.
1. Already talking to payers, there is ‘strong interest’. Already engaged NICE, NHS in England.
Prof. Dollar retweeted
Could not agree more this design is so unethical to HD patients. After all the encouraging rhetoric from the FDA of late, do you feel your communities “patient voice” is being heard and considered by Uniqure and the FDA? Is there an opportunity to influence appropriately?
Prof. Dollar retweeted
Please help @Christina4HD and everyone else suffering from HD by making the BLA review process even faster than priority review of 6 months. @FDA_KyleD please speed up the PDUFA date for @uniQure_NV $QURE AMT130 more in line with the goal of the CNPV program, roughly 2 months from now.
Please don't make them wait any longer than they already have. Also, use flexibility in the design of the confirmatory study. There is no need for a new trial, you can use existing and growing data from the 51 patients already treated upwards of 5+ years ago utilizing this gene therapy and follow the first 100 or so patients who get treated after approval. There are already decades of natural history data to compare efficacy to. Make it happen, for the patients.
@SenRonJohnson
@SenRickScott
@RepAuchincloss
@houmanhemmati
@adamfeuerstein
@armstrongdrew
@l_e_whyte
@temple_west
@laurencurehd
@JRenz0418
@rachelreising96
@geneveritas
@ScottGottliebMD
@BeckyQuick
@davidfaber
@AllysiaFinley
@bradloncar
@docrodwong
@LuckyPenguin10
@POTUS
@SenateAging
@SenBillCassidy
@SenRandPaul
@WhiteHouse
@SusieWiles
@FDACBER
@FDA_KyleD
@DrOzCMS
@HHSGov
#Duchenne
#PrimaryImmunodeficiency
#RareDisease
#HuntingtonsDisease
#FDA #PatientAdvocacy
#PatientVoice
#TimeMatters
#RareDiseaseResearch
#RareDiseaseFramework
@RickSantorum
@FoxNews
@bariweiss
@60Minutes
@CNBC
@WSJopinion
@WSJ
@statnews
Today, the @FDA granted Priority Review to @uniQure_NV AMT-130, bringing the first potential disease-modifying therapy for Huntington’s disease one step closer to patients.
I learned this news while my father lay in the ICU, on a ventilator, with aspiration-related pneumonia.
Pneumonia is the leading cause of death in Huntington’s disease. My father is now confronting one of the very complications that make this disease so devastating.
That is what the irreversible cost of waiting looks like.
A few years ago, he was still running the business he built over 30 years ago. An accomplished businessman, inventor, and published author. Today, Huntington’s disease has taken his independence, his ability to swallow, much of his ability to communicate, and now threatens his life.
My grandmother was diagnosed with HD in 1999. She died in 2011. Twenty-seven years after her diagnosis, we still have no FDA-approved treatment that slows this devastating disease.
As a little girl, I watched HD take my grandmother. Today, my daughter, named in her honor, is watching the same disease take her grandfather and great-aunt.
I am profoundly grateful for today's milestone. But I cannot ignore the human cost of getting here.
I welcome the FDA's decision to grant Priority Review to AMT-130 and strongly support its consideration under the Accelerated Approval pathway. Our community has contributed decades of natural-history research, patient-experience data, and evidence of our willingness to accept meaningful risk.
Every day of delay carries consequences that no subsequent approval can undo.
Years ago, my father asked me, “Christina, what are you doing to leave the world a better place?”
Dad, this is my answer.
I'm fighting for you, for our family, for the more than 41,000 Americans living with Huntington’s disease, and the 200,000+ at risk of inheriting it.
I'm fighting for a future where my daughter never has to stand before the FDA, pleading for a treatment to save her mother, her brother, her aunts, or another generation of our family.
I want her to inherit my grandmother's name.
Not her fate. And certainly not this fight.
💙💜
#HuntingtonsDisease
#RareDisease
#CureHD
Prof. Dollar retweeted
While I'm excited about today's announcement from uniQure, I have very mixed emotions.
I believe AMT-130 works. I believe in its potential to change the future of Huntington's disease. But the road to this point has been an emotional roller coaster for our community, and we're still nowhere near the finish line.
I've lost count of how many advocates and families have asked me, "Why is it so hard for us to get this treatment?"
And the heartbreaking part is that I don't have an answer. Because it shouldn't be this hard.
I'm relieved that the FDA has finally accepted the application for Priority Review, and I welcome the opportunity for an Advisory Committee to examine the evidence and hear directly from patients and families.
However, I still have serious concerns about the proposed confirmatory trial, which I plan to address separately.
We are not asking for shortcuts. We are asking for a regulatory pathway that recognizes the reality of a relentlessly progressive disease and the human cost of every delay.
I'm also deeply disappointed by uniQure's communication with the very community that has fought so hard to get us here. Patients and families deserve transparency, consistency, and respect throughout this process.
But despite all the frustration, today is a step in the right direction.
And as I've said before, we have to allow ourselves to celebrate the wins along the way.
So today, I'll celebrate this progress. Tomorrow, I'll get back up, dust myself off, and continue fighting.
Because progress deserves to be celebrated, but our fight is far from over.
And until we have meaningful treatments available to the people who desperately need them, we cannot afford to stop.
#HuntingtonsDisease #raredisease #TimeMatters #ProgressionDoesntPause
@FDA_KyleD @FDA @FDADrugs @uniQure_NV
"uniQure Announces FDA Acceptance and Priority Review of the Biologics License Application for ifezuntirgene inilparvovec for the Treatment of #HuntingtonsDisease" Check out the full press release on the Huntington's Post! #HDResearch
help4hd.org/post/uniqure-ann…
✨️ $QURE Announces FDA Acceptance and Priority Review of the BLA for ifezuntirgene inilparvovec (#AMT130) for the Treatment of #Huntington’s Disease
Source: uniqure.gcs-web.com/news-rel…
$CLPT $XBI
Prof. Dollar retweeted
For who did not/could not watch **Rare Disease Framework Initiative Hybrid Public Meeting**
October 28, 2026 #HD, #RareDisease
@laurencurehd @Christina4HD $qure
“The final frontier is not space. It’s the fight against neurodegenerative disease.”
Farewell, Captain Picard. 🖖
Perhaps one day, the $QURE Quarians will help write a new chapter, where Huntington’s is no longer a death sentence.
Engage. Make it so. 🚀
tvline.com/2301630/patrick-s…
Prof. Dollar retweeted
I was blessed with the opportunity to speak today at this event. I have the utmost respect for the current FDA administration. You can see and feel how genuine they are, how dedicated they are to this new Rare Disease Framework Initiative. This is some exciting stuff!
Today, FDA kicked off its Rare Disease Framework Initiative with a public meeting, co-hosted by @ReaganUdall. Thanks to all who joined for your input, candor & vision. With only 5% of rare diseases having an approved treatment, the status quo isn't enough — we must do more.
FDA Acting Commissioner @FDA_KyleD shared with the attendees that the “urgency of the need has not changed, but now we — the agency, drug developers, researchers, and patients — are ready to meet that need together.”
Prof. Dollar retweeted
Thank you, Lauren, for representing our community at such a defining moment. Speak with uncompromising conviction. The consequences of regulatory decisions extend far beyond policy and precedent; they determine the futures of entire families. We have watched generations succumb to Huntington’s disease. Now, for the first time, science offers the possibility of changing that trajectory. The urgency of our lives deserves to be reflected in the urgency of the decisions that shape them. 💙💜
Prof. Dollar retweeted
@uniQure_NV @ #ESGCT2026 in Hamburg!
The @ESGCT 33rd Annual Congress takes place in Hamburg, Germany, from 27-30 October 2026.
esgctcongress.com/poster-lis…
$QURE
Prof. Dollar retweeted
Wells Fargo reiterated Overweight on $QURE with a $75 PT (+240% upside) after hosting top HD KOLs:
• BLA-ready AMT-130 alone makes QURE deeply undervalued
• Favorable FDA path = high approval odds
• AMT-260 (rTLE) adds unpriced optionality
KOL insights below 🧵 (1/4)
💯% confidence in @laurencurehd!
The FDA will seek public input on a new Rare Disease Framework, including innovative trial designs, flexible evidence requirements, innovative statistical methods and putting patients’ voices at the center.
#AMT130 $QURE #AdCom4HD
@reaganudall TNX
❤️ I HAVE SOME EXCITING NEWS TO SHARE!
I am incredibly honored and excited to announce that I've been selected to speak in person at the FDA's Rare Disease Framework Public Meeting on October 28th in Silver Spring, Maryland!
This meeting is especially meaningful because it focuses on the FDA's new initiative and recently released white paper, A New Framework for Rare Disease Review.
For someone who has spent so many years advocating for the Huntington's disease community, this opportunity means more to me than I can adequately put into words.
I'm not just an advocate. I'm gene-positive, someone living with the reality of a progressive, rare brain disease. I've watched this disease take so much from people I love, and I know firsthand what is at stake when we talk about treatment development, regulatory decisions, and the time patients have left.
I've spent nearly two decades fighting for a seat at the table. On October 28th, I'll have the privilege of using that seat to help shape a future where the patient voice is not an afterthought, but a foundation of the process.
And that means everything to me. ❤️
The FDA's new Rare Disease Framework Initiative represents an opportunity to rethink how treatments for rare diseases are developed and reviewed, recognizing that rare diseases present unique challenges that traditional approaches don't always adequately address.
It's an opportunity to discuss what evidence is realistically attainable, how we can improve consistency and predictability in regulatory decisions, and how patients can have a meaningful role throughout the process.
But here's the part I really want everyone to hear:
📣 YOU HAVE A SEAT AT THIS TABLE, TOO!
The FDA is inviting public comments on this initiative, and this is an opportunity for patients, caregivers, families, advocates, researchers, and everyone impacted by rare diseases to provide input.
You don't need a medical degree. You don't need to understand every detail of regulatory science.
💪Your lived experience IS expertise. And your voice deserves to be heard. 💙
📣 HOW TO GET INVOLVED:
📝 Submit a public comment:
regulations.gov/docket/FDA-2…
Docket #: FDA-2026-N-12214
📅 Deadline to submit comments: December 11, 2026
📖 Read the FDA's new white paper and learn about the initiative:
fda.gov/industry/fda-rare-di…
💻 Learn about the October 28th public meeting and how to attend virtually:
fda.gov/news-events/fda-meet…
I'm excited. I'm humbled. And I'm incredibly grateful for this opportunity.
But more than anything, I'm hopeful about what we can accomplish when patients are genuinely included in shaping the systems that determine our futures.
We have an opportunity to make a difference. Let's not waste it.
Because for those of us living with progressive rare diseases, every day matters. Disease progression can cause irreversible damage. And what we stand to lose isn't just time - it's our independence, our abilities, precious moments with our families, and the futures we're fighting so hard to protect.
❤️ Time Matters. And so do our voices.
#RareDisease #HuntingtonsDisease #FDA #PatientAdvocacy #PatientVoice #TimeMatters #RareDiseaseResearch #RareDiseaseFramework
Prof. Dollar retweeted
Yet ANOTHER datapoint indicating $QURE 's AMT-130 has disease modifying effect. Look at the drop off in cUHDRS in those who did not complete Week 101. Data point after data point after data point all point in the same direction.
And, the most notable data point of them all remains the updated 3-year data showing highly stat sig 80% slowing on cUHDRS and the stat sig TFC on both 3 AND 4 year, DESPITE the presence of dropouts in the control arm even at 3 year which of course will bias towards being the worst progressors.
