$qure $clpt Jadis Tillery was at the Uk event today and gave some more context regarding amt 130, her comments, her post was on LinkedIn about the entire event but here was her thoughts on amt 130. The first panel was on Huntington's disease, hosted by Fergus Walsh of the BBC, with Professor Sarah Tabrizi, MD PhD FMedSci FRS and Professor Edward Wild of UCL. Their landmark breakthrough has slowed the progression of Huntington's by 75%. Precision neurosurgery, gene delivery, essentially a one-shot treatment for a brutal inherited disorder. Along the way they made discoveries with applications in other neurodegenerative diseases, Alzheimer's among them. The science is the first miracle. What they spent their airtime on was the second. The work with the MHRA that could see the UK become the first country in the world to license this gene therapy, with pathways then opening to Canada, Australia, New Zealand, Japan and Saudi Arabia. That is the difference between a result in a journal and a treatment in a clinic, and it's a real opportunity for the UK. Fergus asked what the reaction was when the results came in. Ed said he had an absolute explosion of joy. I loved that. The work in this room is almost beyond belief in what it can do, and underneath the precision and the protocols, there was a scientist who couldn't quite contain what it would mean for people living with something this cruel.

$qure $clpt $rgnx Good article

Please participate (link in Christina’s post below): As @peter_mantas has correctly argued… $QURE AMT-130 isn’t just impactful on the Huntington’s disease community… If a therapy like AMT-130 — with this level of efficacy (based on the data at this stage) — doesn’t stand a chance, it will have a chilling effect across the entire biotech ecosystem. There are many reasons to care about this. — $CLPT $XBI

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Yesterday, we spoke to the @FDA about why Huntington’s disease should be considered a national priority under the CNPV Pilot Program. Now the FDA is asking for public comments, and our community has an opportunity to be heard. Please take a few minutes to submit a comment asking the FDA to prioritize fatal, progressive neurodegenerative diseases like Huntington’s when promising therapies are ready for review. Comment here: regulations.gov/document/FDA… Comments close June 29 at 11:59 PM ET. #HuntingtonsDisease #HDCommunity #RareDisease

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$qure Powerful testimony today from Huntington's advocates at CNPV

$QURE From FDAs publication today on Real World Evidence (RWE) as Substantial Evidence of Effectiveness (SEE).

The most egregious element of what happened is that if $QURE AMT-130 works, what Makary and Prasad did was give a death sentence to many HD patients that will be knocked out of the time window to receive the treatment. At this point, it’s irrefutable that there’s enough in the data that suggests it *could* work. To call it a “failed therapy” is unconscionable.

Katy, I appreciate your loyalty to Dr. Makary and I truly believe he wanted to reform a broken system. But as someone from the Huntington’s disease community, I need people to understand why so many families were deeply hurt by how we were characterized. We were not “Big Pharma.” We were not paid lobbyists. We were families drowning in generational trauma because our loved ones are dying from one of the cruelest neurodegenerative diseases on earth. And the hardest part? While many Huntington’s families, caregivers, and at-risk individuals were literally on Capitol Hill during Rare Disease Week meeting with lawmakers after paying our own way there, Dr. Makary was simultaneously speaking to reporters about “moneyed interests” and outside pressure surrounding AMT-130. There were no corporate-funded advocacy campaigns behind us. Just families carrying decades of trauma, grief, and fear into congressional offices because we are terrified of running out of time. Nearly 50,000 signatures were gathered from HD families, grassroots advocates, caregivers, and supporters demanding urgency for AMT-130, and those petitions were hand-delivered directly to the FDA by members of the Huntington’s community themselves. Over 10,000 emails were also sent to FDA and members of Congress. Nobody paid us to do that. Fear did. Love did. Desperation did. At the 2024 EL-PFDD meeting in Maryland, FDA sat across from more than 100 HD families and listened as we described watching parents, grandparents, siblings, and children slowly lose movement, speech, cognition, personality, and independence. People in those rooms cried with us. We were told urgency mattered. Soon after we learned FDA aligned with uniQure on an accelerated pathway, many of us finally felt hope for the first time in our lives. So yes, families became loud. Because this disease does not wait. What also deeply damaged trust was watching Dr. Vinay Prasad speak anonymously to reporters about an active therapy. If someone cannot publicly face the people they serve during moments of crisis and scrutiny, they should not be leading CBER. Leadership requires courage, transparency, and accountability. This is especially true in rare disease communities where every delay carries irreversible consequences. Many of us respected Dr. Prasad intellectually. We respected his focus on scientific rigor. But from the patient/family side, much of what we experienced felt dismissive, detached, and at times unnecessarily combative toward a dying community simply asking to be heard. And Dr. Makary protected that approach far too long. The Huntington’s community is not asking for reckless science. FDA itself asked our community what level of risk we would accept, and nearly three-quarters of respondents said they would accept treatment risks for even a chance at 5 years without progression. Because this disease does not wait for “perfect data.” For families living with Huntington’s, every year means more neurons lost, more memories erased, more independence gone, and more people disappearing in front of us. AMT-130 showed the possibility of dramatically slowing that decline over three years. That is time with children. That is dignity. That is life itself. We are not a lobbying machine. We are human beings trying to stop generations of suffering from continuing. 💙💜 #Huntingtonsdisease

$qure $clpt The UK is not messing around The MHRA launches public consultation on new framework that could lead to earlier licensing for therapies for up to 3.5 million people in the UK. The MHRA’s proposed new Rare Disease Therapies Regulatory Framework sets out a fundamentally different approach. Rather than requiring developers to meet standards designed for large-population diseases, it would introduce a flexible, risk-proportionate framework that acknowledges the realities of rare disease development – while keeping patient safety at its heart. The new framework will apply to rare conditions affecting not more than 1 in 50,000 people where there are quantifiable barriers to conducting a ‘standard’ clinical trial. Entry to the proposed new pathway would be guided by criteria such as the severity of the disease and unmet need. gov.uk/government/news/landm…

We are ready to collaborate. We are ready to partner. We are ready to bring meaningful treatments to the HD Community. @Help4HDI @HDSA @HDYOFeed @laurencurehd @POTUS @FDA_KyleD @FDACBER @FDA @RobertKennedyJr @HHSGov

@POTUS @realDonaldTrump @WhiteHouse @US_FDA @FDACBER @FDACDERDirector

While we relax over the weekend celebrating the news of the FDA firings, I thought I would share my recap of the @uniQure_NV $QURE AMT130 study details again. By the end of March 2026, 9 patients in hi dose cohort reached at least 4 years. That will go to 12 in early July. I mention this because in past years, the company reported data in late June/early July using the March cutoff of 9 patients. Could they use these 9 patients when meeting with FDA in upcoming Type B meeting as added support to go back to prior alignment and allow BLA filing ASAP? It is almost criminal not to. We also know that in Sept they released 3-year data on 12 patients and an additional 3 patients did not yet hit the 3-year mark as of that July 2025 cutoff. What if they hit that by now? They can add data from those additional 3 patients in the 3-year data to further support the 3-year efficacy. No later than the end of this year, there will be a total of 20 patients in the hi dose who hit at least 3 years with others hitting up to 5 years. The # hitting 5 years will be very small but this shows the maturity of the data. This is the only treatment for HD to demonstrate any efficacy in all the trials over the years. With a total of 32 patients who have been treated in the hi dose to date, why would new FDA leadership need a phase 3 trial before allowing BLA filing? They should allow filing and if the data package supports AA, approve AMT130 with requirements to continue following all patients in original 4 cohorts plus data on a certain # of patients after approval to grant full approval if they feel it is necessary. Other gene therapy treatments have been granted full approval on fewer than 32 patients. Why delay access to the ONLY disease modifying treatment ever? This is the very definition of a HUGE UNMET NEED and US citizens deserve access before more brain cells die prematurely that will never come back. Hopefully new FDA leadership recognizes the harm done over the past 8 months and reverses course ASAP. Congress, please don't allow the FDA to continue this horror show. Force them to allow uniQure to file BLA and give the data a fair review for AA for the community. @SenRonJohnson @SenRickScott @RepAuchincloss @houmanhemmati @adamfeuerstein @armstrongdrew @l_e_whyte @temple_west @laurencurehd @JRenz0418 @rachelreising96 @BeckyQuick @davidfaber @AllysiaFinley @MariaBartiromo @bradloncar @docrodwong @LuckyPenguin10 @POTUS @SenateAging @SenBillCassidy @SenRandPaul @WhiteHouse @SusieWiles @StevenCheung47 @StevenCheung @Scavino47 @PressSec @SecKennedy @MariaBartiromo @RickSantorum @FoxNews @bariweiss @60Minutes @CNBC @WSJopinion @WSJ @statnews @BillAckman @Loftus @MartinShkreli

AMEN!

$qure Trump today on right to try. Nothing else need to be said. Just common sense. @US_FDA @DrMakaryFDA @laurencurehd @DesertDweller93 @LuckyPenguin10 @peter_mantas

wsj.com/opinion/fda-policies… $qure Gift Link

$qure from John Arnold. Finally some common sense. @LuckyPenguin10 @peter_mantas @DesertDweller93

FDA Policies: Rigorous Science or Roadblocks?$QURE John Arnold in favor of accelerated approvals in gene therapies. wsj.com/opinion/fda-policies…