Craig Reagan retweeted
❤️ 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
Please go to this site and leave comment supporting new rare disease framework and why its vitally important to patients living w/ #ALS, #HUNTINGTONS, #DMD and many others. Its can also an avenue to call them out for intentionally stalling NurOwn!
regulations.gov/docket/FDA-2…
Craig Reagan retweeted
Grateful for @FDA_KyleD's leadership to transform regulatory policy. The #raredisease community desperately needs these regulations to be implemented ASAP.
But while we await this multi-year process, we need @FDADrugs to act wth existing regulatory flexibility to get drugs like #NurOwn, #deramiocel and #AMT130 to people with #ALS #Duchenne and #Huntingtons. People are #DyingWaiting.
#EndALS #EndHD #EndDMD #EndDuchenne
AEI senior fellow @ScottGottliebMD joins CNBC ‘Squawk Box’ to discuss the FDA’s new Rare Disease Framework Initiative and its guidance for developing drugs for unmet patient needs.
Oops, I used the wrong handle last time. Lets try this again... @BrainstormCell
I have a donor that has committed to pay for my NurOwn treatment through the "Right To Try" act. How does the process work? Who do we contact? I'm ready!! #nurownworks
Every word of this is, in reality, far worse than you could ever imagine. Come on, FDA, move your ass!! @SecKennedy @FDA_KyleD @realDonaldTrump @SecRollins
#ALS - this terminal illness is physically, mentally and emotionally draining. The grief of not being able to do what your heart wants. The slow loss of independence. Not being able to fulfill dreams. Each day you lose more and more. The uncertainty weighs heavy.
Craig Reagan retweeted
No child should have to crawl up stairs because his government won't give him access to a cell therapy that could help him retain function.
What are you waiting for @FDA?
@FDA_KyleD, Karim Mikhail and @FDADrugs please approve @capricor's #deramiocel with the same urgency as you did the pancreatic cancer drug that only extended survival by 6 months. The boys and young men with #Duchenne deserve the same chance at life.
#EndDMD #EndDuchenne #rarediseases
#MuscularDystrophy
@POTUS @SecKennedy @SenRonJohnson @SenRickScott @SenGillibrand @SenateAging
@LeaderJohnThune @SenatorWicker @GOPHELP @RandPaul @SenJohnBarrasso @HouseCommerce
I asked Ryu what it is like to live with #Duchenne and this is what he told me.
“At first it was really hard to understand my diagnosis, and some days it’s still really hard. But God helps me with it everyday.”
15 million American children like him are living with a rare disease. So many are waiting for the FDA approved treatments that could extend their lives or ease the symptoms of their disease. @FDA, what are you waiting for?
@FDA_KyleD @FDADrugs @SecKennedy @HHSGov @realDonaldTrump
Apparently, you need to hear this @SecKennedy @FDA_KyleD @realDonaldTrump . While the FDA dicks around w/whether to approve NurOwn for ALS, 40K Americans suffer, along with 1,000's of family members. APPROVE NUROWN NOW! nurownworks.com @MinoShah
facebook.com/share/v/1E65BWh…
Why can't the FDA figure this out?
@SecKennedy @FDA_KyleD @realDonaldTrump
@HeidiOverton
We are dying a miserable death! nurownworks.com PLEASE HELP
facebook.com/share/v/1BxcWKF…
@Vita_Victoria2 @Ml1969 @Humbl4now @JC75408607 @juan23188549 @MinoShah @momwithwheels
Why doesn't the ALS Association ever deliver hope like this?
Answer: Because THEY SUCK! They ONLY support drugs/therapies from which they receive financial BENEFIT!
facebook.com/share/v/1PR4bSL…
@MinoShah @Ml1969 @Vita_Victoria2 @JC75408607 @juan23188549 @SecKennedy @elonmusk
Craig Reagan retweeted
A 75% slowing in lethal disease progression is a HUGE WIN! And these changes on the bounded scale are validated by a reduction in objective biomarker NfL.
@FDACommissioner @FDA_KyleD @FDADrugs
Listen to the people who know #Huntingtons best -- the expert neurologists and patients who have watched generations of their families die.
Use the regulatory flexibility that Congress mandated and grant accelerated approval of @uniQure_NV's AMT130 as these changes are "reasonably likely to predict a clinically meaningful benefit."
#EndHD $QURE
#neurology #MedTwitter #NeuroTwitter
#DyingWaiting
🧬 uniQure has submitted a BLA to the FDA for accelerated approval of ifezuntirgene inilparvovec in Huntington disease, backed by 3-year data showing 75% slowing of disease progression in the high-dose cohort
🧠 Read more: hubs.li/Q04wv2Kq0
#HuntingtonsDisease #GeneTherapy #Neurology
Craig Reagan retweeted
Such a no brainer that @FDA must listen to the specialists who know the disease the best. Hoping @FDA_KyleD and next @FDACommissioner continue to defer to the experts as they did for Replimune approval decision.
People with ALS, #Huntingtons, #ataxia, #Sanfilippo, #Duchenne and so many other #rarediseases deserve the same expertise and same humanity as extended to cancer patients.
Thanks to author, @MinoShah, a @HopkinsMedicine educated anesthesiologist who has been battling #ALS for 15 years. He's seen so many broken promises by the @FDADrugs.
Craig Reagan retweeted
.@ChrisJohnson28, assigning @MyronRolle as a strategic advisor to the @NFLPA is ONLY something Myron will put on his resume. @JCTretter @ReevesMaybin know this. I pray you direct them to get @FDADrugs @FDA_KyleD @FDA @SecKennedy @RobertKennedyJr to give a damn about #ALS
The Danger to NFL Players:
YOU inspired this! NFL players are supporting you, unaware they could be next. There is 4X THE RISK for players, while the public's #ALS lifetime risk is 1 out of every 300 people & is becoming more common (expected to increase at least 40% in the next decade).
A Broken System:
Every new @FDAadministration & Congress give EMPTY PROMISES to a new generation of dying #ALS patients, but we're forgotten the second we walk out. They have 0 ACCOUNTABILITY, because we ALL DIE! I've emailed #FDA leadership & Congress directly thinking that, as a physician who now faces a 100% FATAL death sentence, they might listen when I explain that the @FDACRUEL regulatory rigidity for ALS is not only cruel for the people who are dying now, but it prevents R&D into future treatments from pharmaceutical companies. I WAS WRONG.
The Call to Action:
NFL players going to the #FDA & Congress are things the media and the FDA/Congress can't ignore.
If @FDAdrugs gave a damn about #ALS, you would be getting effective NUROWN, instead of the #FDACBER being nearly 8 months past its own deadline for responding to our NUROWN Citizens Petition.
@realDonaldTrump @DrOz @DrOzCMS @RepGuthrie @RepAndyHarrisMD @PatMcAfeeShow @DeionSanders @AdrianPeterson @MoneyLynch @DeSeanJackson10 @CutonDime25 @AaronDonald97 @markingramII @camjordan94 @VonMiller @CrosbyMaxx @JeromeBettis36 @D_Hest23 @AndrewWhitworth @DBrooks55 @REALPACMAN24 @thereal_lendale @VinceYoung10 @EdgerrinJames @JCTretter @ReevesMaybin @JJWatt @Ochocinco @ShannonSharpe
Craig Reagan retweeted
ACT for ALS has built and supports a robust ALS research infrastructure. Join me in helping to reauthorize it! iamals.org/thank-you-a4a?snw…
Craig Reagan retweeted
Very few people do I respect less than @VPrasadMDMPH.
He has no integrity. It's an awful trait for an @FDADrugs director. People die.
Vinayak lied to the public with the ease & arrogance of a sociopath.
He was the @FDA's grim reaper. There's a lot of blood on his hands, but I don't expect a sociopath to care.
🤖 Made with AI
Craig Reagan retweeted
Very few people do I respect less than @VPrasadMDMPH.
He has no integrity. It's an awful trait for an @FDADrugs director. People die.
Vinayak lied to the public with the ease & arrogance of a sociopath.
He was the @FDA's grim reaper. There's a lot of blood on his hands. I don't expect a sociopath to care.
.@VPrasadMDMPH sits down for the first time since leaving the Trump administration’s @FDA
The WSJ called him the FDA’s Grim Reaper, he says it’s dishonest. This week we talk about why.
He talks to @Getthecheckpod about Moderna’s shares skyrocketing 170%, what the public doesn’t understand about rare disease drugs, and if he identifies as right wing (the answer might surprise you).
00:00 Introducing Vinay Prasad, the FDA's former vaccine chief
04:19 Vinay’s words are moving public markets (a few times!)
10:25 Society weighing education years vs COVID deaths
12:49 How do you run experiments mid-epidemic
17:32 Do masks work
23:38 The ethics of a medical study
26:34 Does Vinay believe in vaccines
37:58 Can a parent say no to cancer treatment for their child
41:27 Lobbyist money in FDA decisions
49:31 What's the FDA's conflict of interest
49:58 Vinay's controversies: WSJ calls him the grim reaper
53:13 The former FDA vaccine chief on @Kalshi’s clinical trial markets
01:00:08 Why is American healthcare so broken
01:01:19 Peptides
01:05:10 Who do patient advocacy groups represent
01:08:49 Vinay supports universal healthcare?!















