LittleFatWillyBarr

[bluefinsashimi @bluefinsashimi]

Hey @US_FDA and @SecKennedy
Patients are still waiting.
Lori Mills just shared her fifth clean brain scan. She’s living proof that ANKTIVA + NK cell therapy works. She testified in front of the FDA months ago. The data has been there. The patients have been there.
Yet the agency continues to drag its feet while people with cancer run out of time.
This isn’t caution. It’s institutional paralysis. Every delay is measured in lives.
Unleash ANKTIVA. Expand access. Stop treating urgency like a suggestion.
Cancer patients don’t have the luxury of your timeline.
@DrPatrick @LoriMills4CA42

[FlyingEagle @FlyingEagle86]

$capr In HOPE‑3, the ITT cardiac analysis included 19 boys who had no objective evidence of cardiomyopathy at baseline which dilutes the ability to see a treatment effect on LVEF over just 12mths. In the pre‑specified subgroup with confirmed cardiomyopathy at baseline (64/83) 1/3

[Jacob @EOWheelsJ]

Repost before it’s too late: Thousands of people like me are about to miss out on an incredible treatment (deramiocel) due to disagreements at the FDA. Please sign this petition to get this drug to the people who need it most.

Lives are on the line!

https://t.co/sMLVojez5f

@cnbc @jimcramer @adamfeuerstein @DC_Draino

[Jesse Brock @JesseBrockVan]

Please stand up for Jacob! Hear his words and show your heart by merely signing his petition

[FlyingEagle @FlyingEagle86]

The pattern is familiar: small rare‑disease trials, strong real‑world outcomes, and an agency that defaults to “no” even when Congress has given it flexibility to consider patient experience and totality of evidence. https://t.co/3NbvhRrHHq 1/2

[RyuStrong_DMD @RyuStrong_DMD]

I'm so grateful to @SenRonJohnson for taking the time to hear me out as a mom to a son with Duchenne muscular dystrophy.

To my son Ryu, a lawmaker who fights for kids with rare diseases is nothing short of a hero.

I was so moved to hear him call out DMD by name during the Senate hearing I attended in February on the @US_FDA's roadblocks to rare disease treatments. Moments like that matter — thank you for seeing families like ours. 🙏

[Lori Mills @LoriMills4CA42]

Everybody, thank you from the bottom of our hearts for your prayers.
We just received our fifth normal brain scan.
Hey FDA: Unleash ANKTIVA and natural killer cell therapy nationwide. Patients are waiting.

MRI BRAIN WO/W CONTRAST 7/31/2026 7:57 PM CLINICAL INDICATION: Neoplasm of the temporal lobe on the right side COMPARISON: None.
TECHNICAL FACTORS: Sagittal T1, Axial DWI, Axial FLAIR, Axial T2, Axial SWI, Axial T1 MPRAGE Post, Sagittal MPR post, Coronal MPR post IMAGING MEDICATIONS: gadobutrol (GADAVIST) 10 mmol/10 mL (1 mmol/mL) injection 10 mL FINDINGS:
Lesion assessment: Prior right temporal craniotomy with residual right temporal convexity dural enhancement. The right temporal resection cavity is again noted.
There was no change in the appearance compared with the prior examination VENTRICULAR SYSTEM: Normal in size and morphology. EXTRAAXIAL SPACES/CORTICAL SULCI: Normal in size and morphology.
MIDLINE SHIFT: None
CEREBRAL PARENCHYMA: No acute infarct.
WHITE MATTER: Normal
HEMORRHAGE: Hemosiderin staining subjacent to craniotomy site.
POSTERIOR FOSSA: Normal
CONTRAST ENHANCEMENT: As above CALVARIUM/SKULL BASE/SCALP: As above VASCULAR SYSTEM: Normal
VISUALIZED PARANASAL SINUSES/MASTOID AIR CELLS: Normal VISUALIZED ORBITS: Normal VISUALIZED UPPER CERVICAL SPINE: Normal
SELLA AND SUPRASELLAR STRUCTURES: Normal
@bullishbruk @DrPatrick @US_FDA

[Bullish @bullishbruk]

$IBRX The ANKTIVA story abroad — across Africa, Saudi Arabia, Qatar, the EU, and Asia is twofold.
The first benefit is clear: geographic expansion and commercial growth.
But the second, and the one most people miss, is even more powerful. Real-world data is being generated at scale. That data will give regulators around the world, including the FDA, the evidence they need to authorize and expand indications faster than anything we’ve seen before. @LoriMills4CA42 @alc2022

[Rachael @Moogsympathizer]

@US_FDA I wasn’t aware that Right to Try was actually “Right to Try” until I was diagnosed with Stage 4 cancer. And now I see all of these cases in feed- people with rare and/ or advanced conditions that have no freedom to pursue treatment. People begging you- the government- over social media to allow them to try a medication. In the USA of all places. What purpose are you serving anymore? @LoriMills4CA42 @realDonaldTrump @SecKennedy

[Jacob @EOWheelsJ]

In light of the committee’s vote against my right to survive, I am sharing my comments submitted to the committee. I encourage all commenters to do the same!

Thank you for the opportunity to provide our perspectives! I am truly grateful for your dedication to the American people. I have lived with Duchenne for 27 years and am not about to stop now. Deramiocel is a drug that can help me continue on my mission to change the world by preserving my heart function. Protecting my heart will give me the opportunity to marry the love of my life, raise the family I deserve, and become the entrepreneur and philanthropist this world needs. These are things that would be impossible without modern medicine.

If Deramiocal is not approved, there’s no other pathway to access this drug that will give me life. Unfortunately, my previous attempts to access it under Right to Try were denied. My cardiologist recently said that he always thinks of me because I never take no for an answer. As much as it pains me to say this, another denial is very likely my last hurrah.

Members of the committee, I ask that you make yes the answer.

@RobertKennedyJr @SecKennedy @FDA_KyleD @adamfeuerstein @POTUS @houmanhemmati @realDonaldTrump @MELANIATRUMP @IvankaTrump @SusieWiles47 @StefanieSpear @elonmusk $CAPR @Capricor #deramiocel @FoxNews @CNBC @jimcramer @business

[Jesse Brock @JesseBrockVan]

FDA is letting down those with rare diseases. Deramiocel $CAPR needs to be approved now! This drug works and there are no other options!

https://t.co/vdPglHIlWB

[Elijah Stacy @Elijahjstacy]

Meeting with Senator Ron Johnson’s office tomorrow! Keep Hope Alive! 🇺🇸

[RyuStrong_DMD @RyuStrong_DMD]

If a treatment is safe, the FDA should let rare disease patients who are out of options make this decision with their doctors.

I am fighting for a chance for my family, my son, and his medical team to decide what treatment is right for him. It should not be a bureaucrat behind a desk in Washington making that call.

[FlyingEagle @FlyingEagle86]

$capr https://t.co/GORNFPtPEh
FDA’s June 2026 draft on“Substantial Evidence of Effectiveness” says: in life‑threatening rare diseases with no options, they should use regulatory flexibility 1 adequate trial + confirmatory evidence can be enough; success criteria & p‑values 1/3

[AZ Latina ☮️🌊🟦 @aVoice4ALS]

Hey @US_FDA...
This video should be part of your risk benefit decision re @capricor's #deramiocel.

People without terminal rare diseases have NO comprehension about what it takes to live each and every day.

Consider the risk of a Type II error. If FDA does nothing, the toll of #Duchenne gets worse. If you grant accelerated approval, science wins and her son gets a chance to preserve his upper limb function and maybe heart function too.

If this were your son, you would want that chance.

@FDA_KyleD @FDACBER @FDACommissioner
@POTUS @SusieWiles47 @SenRonJohnson @SecKennedy

#EndDuchenne #EndDMD
#DrugsInBodies

[Jesse Brock @JesseBrockVan]

This is the face of DMD. @US_FDA @Capricor @SenRonJohnson @RobertKennedyJr Approve Deramiocel!

[FlyingEagle @FlyingEagle86]

The irony speaks for itself.@FDA_KyleD @FDA_KyleD @HHSGov @RobertKennedyJr please approve @Capricor ‘s Deramiocel & improve these young men’s quality of life. The risk/benefit is like no other, listen to the patients,families, and care givers. #dmd @ParentProjectMD @CoalitionDMD

[RyuStrong_DMD @RyuStrong_DMD]

What does it take to keep my son alive? Each year it seems we add more equipment. 

This is the reality of Duchenne. And it is why we need the @US_FDA to approve Ryu’s treatment so he hopefully will not need all of this equipment, at least not any additional pieces. #ApproveHopeNow

[Mary B Schanck @MaryBschanck]

$CAPR I strongly disagreed with the comments suggesting that increasing LVEF would be detrimental to DMD patients. In my view, those statements had the potential to mislead the advisory committee during an important discussion.

I’ve shared my concerns with FDA CBER leadership and requested that they review the matter carefully. I also reached out to Linda to encourage a public response addressing what I believe are inaccuracies. If you feel similarly, I encourage you to respectfully submit your own comments to the appropriate channels. Thoughtful, evidence-based feedback is far more likely to have an impact than personal attacks.

Patients deserve decisions based on the best available science and an objective evaluation of the evidence.

[Ja Hish @YaKhmarMajnoon]

$CAPR
FDA AdCom on Jesus:
“Wounds look promising, but p>0.05.”

Thomas: “I need to see the data.”

Jesus: “I’m right here.”

Scientific purists: “Insufficient follow-up.”

FDA: “CRL.”

Maybe they just didn’t give him enough time to prove himself.