Ryan @Ryan4HD

Signed by myself and a couple others in the rare disease community💙 honored to represent multiple rare diseases @POTUS @realDonaldTrump @RFKJr_Official realclearhealth.com/blog/2026/05/1…

@SenRonJohnson Please address the topic below re Huntington's Disease and @US_FDA reversal of uniQure AMT-130.

The AMT-130 fight isn't happening in isolation. Regenxbio's Hunter syndrome gene therapy: rejected. Capricor's Duchenne cell therapy: rejected. Biohaven's spinocerebellar ataxia treatment: rejected. Moderna's mRNA flu vaccine: initially refused, reversed after White House pressure. Every one of these happened under the same CBER leadership, in the same six-month window. This isn't about one drug. This is a pattern. And until the system changes, every rare disease community is one anonymous press call away from the same nightmare. #RareDisease #FDA #PatientAdvocacy

With CBER director Vinay Prasad set to depart the agency at the end of the month, a coalition of patient groups and biotech executives penned a letter imploring the Trump administration to “restore regulatory clarity” for rare disease therapies. Experts on a BioSpace panel last week also acknowledged the challenges faced by a more stringent FDA. #CBER #biotech #regulatory #rarediseases #biospace hubs.li/Q049qQbT0

Here's why the 48-month AMT-130 data matters so much. At 36 months: 75% slowing of disease progression, p = .003, CSF NfL below baseline. Gene therapy is designed to be durable. If the effect is holding or strengthening at 48 months, it answers the FDA's biggest remaining question: is this a real, sustained disease modification or a temporary signal? Four years of follow-up data in a neurodegenerative disease is rare. This dataset will be one of the longest in Huntington's research history. Expected Q3 2026. Mark your calendar. $QURE #HuntingtonsDisease #AMT130

@SenateAging @SenRickScott Senator Scott, please help the Huntington’s disease community - the FDA is rejecting life saving therapies and demanding phase 3 placebos which our loved ones do not have the time for. We need your help. @SenRonJohnson @GraceCarnathan @SusieWiles @KushDesai47 @LauraLoomer

Thank you Angela Smith Wilkinson for allowing us to share Awareness for Huntington's Disease at Copiah-Lincoln Community College today #WilmothWarriors

The Right to Try Act was signed in 2018. It was supposed to give terminally ill patients access to experimental therapies. In practice, almost nobody gets access through it. Pharma companies are reluctant to provide drugs outside trials. Most therapies haven't passed Phase I yet. The paperwork is a maze. Early-stage clinical trials are the real right to try. And the FDA is making them harder, slower, and more expensive every year. If you care about patient access, the clinical trial system is where the fight is. Not just the approval decision at the end. #RareDisease #RightToTry #PatientAdvocacy

quick chat with the Stat team about rare disease and fda. adam asked what i had to say about those who argue vinay brought rigor back to fda and that patient advocates had too much influence. what i wish i articulated better is what people who have been involved in rare disease trials will tell you is even if u have a cure, u often can't run a gold standard randomized trial to prove it if a disease is too rare, heterogenous, or slow. patient advocacy leaders understand this intimately well and regulators would make better decisions if they listen more to them rather than less.

canoebrookbl @canoebrookbl

3 months ago

Readout LOUD podcast: biotech investors' plea to Trump. ⁦@docrodwong⁩ thanks Rod for speaking up on behalf of the rare disease community! statnews.com/2026/04/02/rea…

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Have to admit I’d be quite mad if I was a patient with HD and saw MM touting how fast they are moving to approve new drugs for a disease where you already have multiple drugs already approved and which can be mitigated by lifestyle intervention…

$qure $clpt Heck of a story on what families go through in their battles with HD, highly recommend this article. Heather, we will keep fighting also. Really heartbreaking stuff. The HD community is asking for a fair shot, for their story to be told, keep fighting my friends. It makes your skin boil when the guy that turned down the program didn’t even bother to show up to the Type A meeting and hear out a person like Victor Sung. After the meeting, he is going to shit on the program? That is sickening behavior. Karma is a real thing in this world and Prasad will have his judgment day. The person that has seen the most hd patients with amt-130 (Sung) and their results in the entire world, that is why people are so pissed. That type of behavior is repulsive, at least hear people like that out. They deserve an audience, when Dr. Woodcock says it’s truly evil, you stand up and fight. She has nothing on the line, one of the most respected prior FDA officials saying that, you know something is very wrong here and the HD community should be at the White House and FDA’s front door until they get their fair shot. “But fight we do, and continue to fight we will. For our at-risk loved ones, and the ones who are pre-symptomatic, mid-stage, late-stage, and even after.” “We fight because we want to help our loved ones; we don’t want others to go through what we are currently going through. We want that understanding, we want to spend our lives with our loved ones. We want that awareness… but ultimately… We fight for that treatment, and we fight even harder to have a cure.” “It’s watching the decline of my husband. Going from a hardworking, present father who loved to have fun, to someone who can’t walk without help, can’t speak clearly, and needs help in daily life.” patientworthy.com/2026/03/28/hea… @hthurgood @RepAuchincloss @SenRonJohnson @SenRickScott @SenGillibrand @WhiteHouse @FrankLuntz @SusieWiles @BeckyQuick @HDSA @HDBuzzFeed @Help4HDI @adamfeuerstein @MartinShkreli

Let's put a face to this story. This is @hthurgood's husband Nathan. #huntingtonsdisease #therealhd #thishdlife @SenateAging @US_FDA @FDACBER @fdacder

@laurencurehd @Help4HDI There is real hope to slow Huntington’s, but hope means nothing without action. The @US_FDA must urgently back therapies like AMT-130. Every delay prolongs suffering for families who don’t have time to wait. @DrMakaryFDA, history is watching. What happens next is your choice.

I’m seriously considering doing a series on #HuntingtonsDisease to show what we mean when we say #ProgressionDoesntPause because too many people still don’t fully understand what that looks like. 🧵 (1)

People ask me why I care so much about Huntington's disease. It's the cruelest math in medicine. If one of your parents has it, you have a 50% chance. You can get tested and know your fate, or you can live with the uncertainty. There's no right answer. Some families have three generations affected at the same time. A grandmother in late stage. A parent losing motor function. A child who just tested positive and is counting the years until symptoms start. And for all of them, the answer from medicine has been the same for decades: there's nothing we can do. AMT-130 is the first therapy that might change that sentence. That's why this fight matters. #HuntingtonsDisease @Help4HDI

Alterations of brain-derived neurotrophic factor, nerve growth factor and neurotrophin-3 levels across biofluids and brain regions in Huntington's disease: A comprehensive systematic review, meta-analysis and meta-regression of human and rodent studies. dlvr.it/TRl6kp

100 days from surgery to raiding World of Warcraft with pure thought control. This is the pace of innovation when patients actually get access. Incredible stuff. Stories like this are why I fight for Huntington's disease families. The science is there. The technology is there. The gap between what's possible and what patients can access is the only thing that needs to catch up.

Jon L. Noble🇬🇧 @CheckCanopy

4 months ago

It’s hard to believe it’s already been 100 days since I received my Neuralink N1 implant. Looking back, the whole journey feels like science fiction that somehow became my everyday reality. The surgery on Day 0 was surprisingly easy. A quick general anaesthetic, a small

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PLEASE pay attention to this! The #huntingtonsdisease community - the #raredisease community as a whole - needs your help, @SenRonJohnson @SenGillibrand @SenRickScott @SenateAging @SenTedCruz @RepAuchincloss @WhiteHouse @realDonaldTrump @BeckyQuick @HHSGov #saverare #DelayStealsTime #ActWithUrgency #HDLivesOnTheLine

CP2 @LuckyPenguin10

4 months ago

🤔From the FDA's public calendar: On Mar 3, 2026, FDA Commissioner Makary met with "Arnold Ventures representatives" at FDA headquarters in Silver Spring, MD. Multiple FDA staff attended. [1] | Subject: "Collaboration Opportunities" Here's what was happening that same week: —

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@MAHA_Action My understanding is uniQure says #Huntingtonsdisease slowed by 75 percent but #FDA asking for brain surgery placebo. Multiple spinal taps the patient may advanced and not qualify. Unethical We already have a wonderful natural history study Enroll HD. Please approve AMT-130.

@US_FDA #UniQure says AMT-130 slows #Huntingtonsdisease by 75 percent but you haven't approved. Huntington’s disease is the worst illness known to mankind and has nothing.