KL (for HD)

After calling the Huntington’s disease community the “swamp”, @AndrewNixonHHS blocked me on X for commenting on his posts asking why the FDA was rejecting the first disease modifying therapy that slows the progression of Huntington’s disease - a rare and deadly disease that affects 200,000 Americans. A few key takeaways: 1) Andrew Nixon is a 27 year old kid who is impulsive in his comments and a significant liability to the Trump administration. We must continue to tag @StevenCheung47 @DanScavino @SusieWiles @WhiteHouse @SenRonJohnson @SenRickScott @RFKJr_Official @SenRandPaul - to let them know what @DrMakaryFDA is doing at the FDA. The most unpopular thing the FDA can do a few months before the midterms is to reject a life saving therapy that affects 200,000 Americans AND their loved ones while there currently are no alternatives. President Trump should be championing this therapy (called AMT-130 by @uniQure_NV) and taking credit for accelerating the approval of rare diseases and yet Makary and Prasad has made the FDA a source of embarrassment. Their cruelty has inflected untold pain to the average American. Please also tag the media. There must be stories on this: @BeckyQuick @60Minutes @CBSNews @GMA @FoxNews @FrankLuntz @WSJ @WSJhealth @carlquintanilla @andersoncooper @foxandfriends @joerogan @TheStalwart @business We must put maximum pressure so that Makary’s arrogance and cruelty are clearly exposed to the Trump administration. @Christina4HD @laurencurehd @HDSApacific @HDSA @HDSA_SF @ingentium_hd @HDA_tweeting @JRenz0418 @mike98572986 Please share this as much as possible! Collectively we can make a difference. @POTUS - we trust that you’ll help us. We believed in MAHA. Please do not make us regret this decision.

Huntington's gene therapy research has reached a turning point, expert says dlvr.it/TRcW1c

Holy crap, footage from Iran’s Dezful airbase earlier today. American and Israeli missiles are another level Turn on the audio

It’s getting hard to call this “being careful”. In rare diseases, dragging timelines like this does not protect patients, it just takes away their shot. We’re watching it happen in real time. @DrMakaryFDA $SRPT $QURE

@HHSGov @DrMakaryFDA @WorldDSDay @SecKennedy The Huntington’s disease community needs the FDA to approve AMT-130!!!!

Yesterday, @HHSGov recognized @WorldDSDay by celebrating the lives and resilience of people with Down syndrome. @SecKennedy and @DrMakaryFDA spent meaningful time with local families, underscoring the power of community and our commitment to supporting people with disabilities at every stage of life.

This @US_FDA job posting would be funny if it weren’t so disappointing — and candidly, sad to see the state the FDA has been led to. The CBER Director is a Title 21 position. One of the most consequential regulatory roles in the world. The person in this seat oversees gene therapies, blood products, and cellular therapies for ~330 million Americans. And they're hiring for it like it's a regional sales manager. Here’s what this signals... 🧵[1/8] $QURE $XBI #FDA

@MAHA_Action FDA must approve AMT-130, the Huntington’s disease community will not rest until the fda honors prior guidelines- we cannot wait another 3-4 years - let President Trump take credit for this! Our loved ones depend on it!!

The most powerful health habit might be the simplest one. Just 20 minutes of walking a day can dramatically improve chronic disease and mental health outcomes, according to Mehmet Oz. At the same time, the U.S. Food and Drug Administration is accelerating cancer treatment approvals, with some therapies showing life-changing results for patients. And new research continues to confirm what many have been warning about: ultra-processed foods are driving serious health risks. High consumption has been linked to a significantly increased risk of heart attack, stroke, and death. Meanwhile, the U.S. Department of Agriculture is investing billions into strengthening rural communities and infrastructure. Small habits. Big shifts. Real impact. @CSinclairtv

If you're just hearing about Huntington's disease for the first time because of the AMT-130 story, here's what families live with every day: A parent watching their child lose the ability to walk, speak, and think. Knowing their other children might carry the same gene. Knowing there's no treatment. Watching it happen twice, sometimes three times, in the same family. That's who the FDA called "moneyed interests." That's who an anonymous official dismissed while calling the only promising therapy "snake oil." These families aren't investors. They're parents doing math on how many years their kids have left. Follow @Help4HDI. The fight continues. #HuntingtonsDisease #AMT130 #RareDisease

@WhiteHouse @SusieWiles @StevenCheung47 @Scavino47 @SenRonJohnson - The Huntington’s disease community needs your help. This disease affects too many Americans for the FDA to reject a treatment and demand a phase 3 placebo trial that would take years.

Getting sent around in BBG chats re $QURE

"the science is cumulative, and the cumulative weight here is becoming extraordinary." $QURE

@HHSGov APPROVE AMT-130! Huntington’s disease patients cannot wait several years. Let President trump take the credit for his work accelerating approvals of treatments for rare diseases!!! @SenRonJohnson

⬇️ hhs.gov/nutrition-educ…

HHS and Ed. are tackling chronic disease head-on by elevating nutrition education at medical schools.

CBER's 'gold standard' for #AMT130 $QURE? Double-blind RCT with sham brain surgery, drilling holes in skulls while HD patients go from dancing with family to dead in 4 years. Cruel. Unethical. Prasad and Szarama don't give a single shit about suffering patients. This isn't science, it's sadistic gatekeeping. #TimeMatters #Iamtheswamp #Huntingtons @US_FDA

I would like to offer to pay the salaries of TSA personnel during this funding impasse that is negatively affecting the lives of so many Americans at airports throughout the country

@sarahdespres @SecKennedy @DrMakaryFDA And yet the FDA rejects cell and gene therapies for Huntington’s disease, DMD, Hunter syndrome, etc.

Despite a dearth of data to prove these products work and aren’t dangerous, @SecKennedy and @DrMakaryFDA poised to expand access to unapproved, injectable drugs that are the darling of wellness influencers. This ends well. subscriber.politicopro.com/article/2026/0…

@WhiteHouse @StevenCheung47 @Scavino47 @SusieWiles @SenRonJohnson @SenRickScott Please ask the FDA to approve AMT-130 by Uniqure, the first disease slowing gene therapy for Huntington’s disease. Makary and Prasad have demanded a phase 3 placebo trial and our loved ones cannot wait another 3-4 years. We believed in MAHA - please help us!! @MariaBartiromo @FoxNews @BeckyQuick @andrewrsorkin @SullyCNBC @BillAckman @SenBillCassidy @LauraLoomer @SenateAging @HDSA

@HarrieScarlet Good point - and w the measles outbreak and handful of other gaffes, hoping there’s change

Kristi Noem was a loyalist too. Remember Trump doesn’t like liabilities and canning RFK Jr doesn’t mean total ostracism. He will get promoted to be something else

Katie Jackson's husband died from Huntington's disease. Her three children each have a 50% chance of inheriting the gene. She runs Help 4 HD International. She delivered 48,000 petition signatures to the FDA. She called the sham surgery demand unethical. When the FDA's anonymous official was trashing AMT-130 from behind a press call, Katie was standing in front of cameras with her name on everything she said. That's the difference between courage and bureaucracy. @Help4HD #HuntingtonsDisease