Rae Tennant

[Lauren Holder @laurencurehd]

I also appreciate the detailed explanation because it's difficult to get all of that across to other advocates. So thanks for making that part easier.

I also respect you greatly. You have fought alongside us through this and I am grateful beyond measure.

However, I hope everyone remembers that we are fighting for our lives. THIS confirmatory trial design isn't fighting for our lives. It will 100% cause progression of disease and irreversible damage for not 1 year, not 18 months or 2 years but THREE years. It doesn’t matter where they are located, that is unethical and wrong when we KNOW what this treatment does, when other regulatory bodies are not requiring the same thing because of how unethical it is. And we have the right to be angry because we have ALREADY been fighting this fight, and it feels like NO ONE IS LISTENING.

Another thing - right now IS a historic time for our community because for the first time EVER, WE HAVE OPTIONS! So - if no one is listening, if the KOL aren't fighting for something better because of what we as patients have been saying, why are we fighting so hard when they're okay with sacrificing brain function and ultimately lives with a trial design like this??

[Lauren Holder @laurencurehd]

Please take the time to read the photo - it's enlightening and shows we still have a long way to go in the #raredisease communities. My thoughts? Let's fight this together. Enough is enough.

[Lauren Holder @laurencurehd]

💯 this. This isn't okay. This isn't better than before. The wording just changed - the requirements for the confirmatory trial are unrealistic and still unethical.

Just because the "sham" surgery isn't physically being done doesn't make this better. A "sham" or "placebo" arm is being required where the participants would not be able to receive the actual treatment or participate in any other research for THREE YEARS. Progression in that time would cause irreversible damage to the brain and most likely keep us from being able to participate in any future research.

And to require 200 participants in the USA for a rare disease like HD - my mind is blown by all of this. Yes, time for some serious discussion and advocacy.
@FDA_KyleD @FDA @FDACBER @fdacder @WhiteHouse

[Prof. Dollar @Prof_Dollar]

$QURE “I don’t think we need a cure,” neuroscientist said. “If you can slow it down by 50 percent and pick people up early on, you’ve probably done it.”
#AMT130 reported ~75%.
He gets it.
Patients gets it.
Only question left:
Does the FDA get it?
https://t.co/vBBoWpoYRL $CLPT

[Lauren Holder @laurencurehd]

Truly incredible ❤️ For someone with #huntingtonsdisease to be able to go back to work after being "medically disabled" - it's a first in our community. This is a game changer.
#raredisease #AMT130 #GeneTherapy @US_FDA @FDA_KyleD @FDACBER @FDACDERDirector @fdacder @Help4HDI @WhiteHouse @POTUS @realDonaldTrump

[Dr. Toonces, MD, PhD @DrToonces]

The thing that gets lost in the regulatory back-and-forth: Huntington's doesn't pause while the paperwork moves.

Every month of delay is a month of progression for families who don't get those months back. The clock the agency runs is the same clock the patients are losing.

$QURE #AMT130

[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

[Lauren Holder @laurencurehd]

Congratulations to Dr. Sarah Tabrizi! This recognition is so incredibly well deserved.

I’ve had the privilege of knowing Dr. Tabrizi and following her work for many years, and her impact on the #huntingtonsdisease community cannot be overstated. Her research has brought hope and meaningful progress to families facing HD, but what sets her apart is the way she partners with our community. She doesn't just work for us - she works with us, listening to our experiences, valuing our perspectives, and standing alongside us every step of the way.

Dr. Tabrizi embodies what true collaboration looks like: Scientists and patients working together toward a shared goal.

Dr. Tabrizi, thank you for your dedication, your compassion, and your unwavering commitment to improving the lives of people and families affected by HD. The HD community is better because of your leadership, and I’m grateful to have witnessed your impact firsthand. 💙
#raredisease #Voice4HD #hdresearch #HDWarrior
@FDA_KyleD
@US_FDA
@houmanhemmati
@Help4HDI
@HDYOFeed
@Christina4HD
@JRenz0418
@hthurgood
@rachelreising96
@BrittanyMegan10
@peter_mantas

Link: https://t.co/IpgOKWyeDe

[DesertDweller_ROAR_4_A_CURE_4_HD @DesertDweller93]

@mike98572986 Must be the dreaded placebo effect, NOT!!!

How can anyone disagree that @uniQure_NV $QURE AMT130 should get approval to file BLA? This is criminal.

Mike, you are the best, uncovering all these articles and evidence to support my belief that the FDA will realign shortly.

[Lauren Holder @laurencurehd]

Dr. Victor Sung is an incredible doctor. He is highly respected in the #huntingtonsdisease community. As one of the actual doctors to treat patients with @uniQure_NV
#AMT130 AND follow them long-term, what he says carries weight.
But more importantly, at least to me, he acknowledges what we have been saying as patients/participants:

"Even with those constraints, Sung said, the patient community’s response to the FDA decision has been clear. "If I could summarize their stance, it has been ‘let us be the arbiter of our own risk tolerance,’” he said.
#raredisease
#ActWithUrgency #TimeMatters #DelayStealsTime
@RepAuchincloss @SenRonJohnson @SenRickScott @SenateAging @adamfeuerstein @houmanhemmati @l_e_whyte @peter_mantas @DrToonces @statnews @WSJ @Bloomberg @FDA_KyleD @FDACBER @POTUS @WhiteHouse @realDonaldTrump @US_FDA @help4hdi
Original article here: https://t.co/nXDQos2aLp

[Dr. Toonces, MD, PhD @DrToonces]

The part that's easy to miss about Huntington's: there has never been a treatment that slows it. Everything approved manages symptoms while the disease keeps moving underneath.

That's the bar AMT-130 is trying to clear. Not symptom relief. Disease modification.

$QURE #AMT130

[DesertDweller_ROAR_4_A_CURE_4_HD @DesertDweller93]

Fantastic and heart wrenching testimony from @Christina4HD at today's FDA meeting on CNPV. Let's hope the members of the @US_FDA understand the history and the absolutely disgraceful decisions by prior leadership and grant a CNPV to @uniQure_NV $QURE AMT130.

[DesertDweller_ROAR_4_A_CURE_4_HD @DesertDweller93]

Thanks Christina and Karl for doing this for the community.

Give @uniQure_NV $QURE AMT130 a chance and allow them to file and grant them priority review in an attempt to remedy the travesty caused by @VPrasadMDMPH & @DrMakaryFDA that denied access to this vulnerable population.

@SenRonJohnson
@SenRickScott
@RepAuchincloss
@FDA_KyleD
@FDACBER
@houmanhemmati
@adamfeuerstein
@armstrongdrew
@l_e_whyte
@temple_west
@laurencurehd
@JRenz0418
@rachelreising96
@ScottGottliebMD
@BeckyQuick
@davidfaber
@AllysiaFinley
@MariaBartiromo
@bradloncar
@docrodwong
@LuckyPenguin10
@POTUS
@SenateAging
@SenBillCassidy
@SenRandPaul
@WhiteHouse
@SusieWiles
@StevenCheung47
@StevenCheung
@SecKennedy
@FDACBER
@FDA_KyleD
@HHSGov
@MariaBartiromo
@RickSantorum
@bariweiss
@60Minutes
@CNBC
@WSJopinion
@WSJ
@statnews
@BillAckman
@Loftus
@MartinShkreli

[Lauren Holder @laurencurehd]

WHY wasn't the #huntingtonsdisease community included in this meeting?!?!

[. @viatoCEO]

@FDA_KyleD are you kidding me?

The reversal on $QURE AMT-130 was a shock to the Huntington’s Disease community and you won’t even give them a seat at the table?

[DesertDweller_ROAR_4_A_CURE_4_HD @DesertDweller93]

Great article and let's hope @FDA_KyleD follows its suggestions, especially the one about remedying poor FDA decisions. This applies to @uniQure_NV $QURE AMT130 BLA filing decision.

Allow uniQure to file BLA and give it a fair review. That is all the HD Community is asking as a remedy.

https://t.co/DpueQnHQoo

[Skyhawk Therapeutics @Skyhawk_Tx]

We are excited to announce this morning the twelve-month Phase 1 / 2 data for Skyhawk’s SKY-0515 small molecule splicing modifier for Huntington’s disease! Press release is here: https://t.co/O1SOImQ2KH
#huntingtonsdisease

[Lauren Holder @laurencurehd]

My heart breaks watching this video. This is the reality of young caregivers of a parent with #huntingtonsdisease. A disease that doesn't affect us when we are older - no, it usually strikes in the prime working years, in the years you are building your life - average age of onset is between 30's and 50's.

No cure. No way to slow it down...oh wait! 🤔 #AMT130 #ActWithUrgency #HDLivesOnTheLine @FDA_KyleD @US_FDA @FDACBER @POTUS @WhiteHouse @realDonaldTrump @SenateAging @SenRonJohnson @SenRickScott @SenGillibrand

https://t.co/GnyTXEJYLM

[canoebrookbl @canoebrookbl]

Job not done yet for $qure, $bhvn. Let us keep the pressure on. https://t.co/3LMmsElLRP

[twotreesinthecenter @twotreesthere]

Thank you Sirs @SecKennedy @FDA_KyleD & @US_FDA @FDACBER for your commitment to bring safe & effective treatments to American patients urgently. $REPL $QURE
Source of screen shot: https://t.co/Nlek8UhbPy