Karen P

longtime biotech investor, tweets are my personal opinion, not investing advice

[Karen P @catt30VA]

@Gantosj I love the amount of investment that will allow Liberty to continue to expand. I wonder what exactly those warrants will amount to in terms of dilution of stock?

[NervGen @NervgenP]

More than 15 million people worldwide live with SCI. Behind every number is a person with individual experiences, priorities and goals.

This #SCIAwarenessMonth, join the conversation and register for the Patient-Focused Drug Development meeting on SCI: https://t.co/TFnu8gIvd8

[Father Jim Sichko @JimSichko]

With ALL my contacts, I’ve been PRIVATELY trying to reach President Trump with ONE request:

🇺🇸 SIGN AN EXECUTIVE ORDER FOR A DOLLY PARTON U.S. POSTAGE STAMP — with proceeds supporting CHILDHOOD LITERACY and the IMAGINATION LIBRARY! 📚❤️

NO ONE will put my call through…

Apparently, they’ve already heard about me! 😂

Mr. President — CALL ME! ☎️🇺🇸

#DollyStamp #DollyParton #ImaginationLibrary #USPS @realDonaldTrump @Pontifex @AndyBeshearKY @RepAndyBarr @SenMcConnell @DollyParton @dollyslibrary

[Antonio Linares @alc2022]

Stacye was left completely paralysed after an accident

Then she took a peptide called NVG-291

A few weeks ago she was able to walk a few blocks with a walker

FDA peptide meeting this week. Watch this before everyone else does

[Masih Alinejad @AlinejadMasih]

STOP comparing Afghan women in burqas and Iranian women under compulsory hijab with Christian nuns.

This really isn’t complicated.

A nun chooses to become a nun. Her vow applies to her. She doesn’t take the vow and suddenly every woman in Italy has to cover her hair.

And if she removes her veil, the morality police don’t show up to beat her, arrest her, or throw her in prison.

That’s the difference.

In Iran and Afghanistan, the rules don’t care whether you’re religious, secular, Muslim, atheist, or just want to be left alone. You’re a woman? Congratulations. The authorities have a dress code for you.

And please stop pointing at little girls in hijab and calling it “choice.” Some of these children can’t even read yet. They’re old enough to choose a religious obligation for life, but apparently not old enough to choose what they want for lunch?

Here’s the test nobody seems to want to take:

What happens when a woman says NO?

If the answer is guns and bullets, prison, beatings, losing your education, or being erased from public life, congratulations, you’ve discovered the difference between choice and coercion.

And if you still think the Taliban model is somehow “liberating,” try it yourself. Cover up. Obey. Let religious authorities decide how you dress, where you go, whether you work, and when you’re allowed to speak. Then explain to Afghan women how empowered you feel.

Is this really that difficult to understand?

#LetUsTalk

[Karen P @catt30VA]

@Elijahjstacy @YoungKimCA @RepYoungKim Your tireless work is greatly appreciated and I'm so happy to hear that your mother is doing well!

[Pete Zerria @PeteZerria]

@adamfeuerstein In rare disease setting PUL data is excellent, just ask The Lancet. Better yet ask the kids who have been getting drug for years, and their families. Highly approvable by FDA. PDUFA extended to 11/20. Conditional approval granted. Not every day that price target raised by 800%.

[Asher77 @Asher3334]

@C5625797576187 The Trump administration has fought for logic in decision making yet old dogma still holds strong.

[Jesse Brock @JesseBrockVan]

Brilliant post Adcom Analysis! Deramiocel must be approved!@Capricor $CAPR @ParentProjectMD @SenRonJohnson @RobertKennedyJr @realDonaldTrump @houmanhemmati
@Elijahjstacy

[CPT_Saki @macau_w]

$CAPR

HOPE 3: Deramiocel outperformed placebo in rank-based scoring. (drug has treatment EFT)

all DMD Trials: fail to show 1-year absolute effect.

HOPE 2 / OLE: 5-year data proves long-term efficacy.

Supported by general DMD natural progression studies.

Deramiocel works.

[FlyingEagle @FlyingEagle86]

Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial - The Lancet https://t.co/j8BvbyclOI

$CAPR #Deramiocel #DMD @US_FDA what more can we ask for please

[Karen P @catt30VA]

@adamfeuerstein Please review the CAPR answer to this erroneous graph at the Adcom to the FDA today. It is based on the 1.1 SAP pre-study projection of a trial. It is not the result if you use the approved study SAP 1.7, and is not born out if you use 2.0 or 3.0 either.

[Karen P @catt30VA]

@AscendingBio @MaryBschanck These three doctors as well as others have presented their educated and first hand observer supportive statements to the FDA today at the Adcom. Well worth the listen.

[Markland55 @markland_55]

1/ $CAPR has been selling off on talk that the company might have been inadvertently unblinded before updating the HOPE-3 statistical plan. That story is getting more attention than it deserves. Here’s the actual situation.
2/ Capricor was very deliberate about this. They told investors back in 2025 that they would send the revised SAP to the FDA first, wait for feedback, and only unblind after that. That’s exactly what they did.
3/ Linda Marban has been clear on this. The company was not unblinded to the results before the SAP was submitted and approved. The changes were discussed with the FDA and they had their blessing. Everything was documented and shared with the agency.
4/ She even said this is probably just something the FDA wants to go over at the AdCom for transparency. Not because there’s an actual issue.
5/ On top of that, the change in how they analyzed the PUL 2.0 data (moving toward relative change and percent slowing instead of just absolute mean change) actually makes the results easier to understand. DMD patients start at very different levels of function, so showing how much the drug slowed decline compared to placebo (54% in this case) is more meaningful than raw point differences alone.
6/ PUL 2.0 itself is a solid, well-established tool. It was developed by an international group of clinicians and researchers, including Anna Mayhew and Eugenio Mercuri, and refined with better statistical methods to track upper limb function in DMD.
7/ The July 29 AdCom is there to look at these exact kinds of questions. The FDA will lay out any concerns they have, Capricor will walk through the timeline and procedures, and the panel will discuss it openly. That’s how these meetings work.
8/ At the end of the day HOPE-3 hit its main goal on PUL 2.0 and the key heart function measure, with results that line up with what they saw before. The noise around unblinding is mostly speculation that doesn’t line up with how the company actually ran the trial.

[Houman David Hemmati, MD, PhD @houmanhemmati]

@MaryBschanck Just came out https://t.co/Wk0mYK0E2J

[Human Shadow @HumanShadow0x]

1/ 🧵 Since yesterday, everyone has been talking about the FDA briefing, the $CAPR @Capricor stock crash, and the market reaction. But behind every chart and every headline, there is something much more important: people, families. Children living with Duchenne muscular dystrophy

[Darp101 @DarpResearch]

Fantastic news. Video of 4 people in $NGEN NervGen drug trial for spinal cord paralysis treatment NVG-291. All on the drug had amazing results. One regained use of her hand in just 3 weeks on the drug. All can mostly walk now. The drug has kept working after they stopped using it; it allows nerves to regrow, and the nerves keep growing and connecting even months after treatment. If anyone can get this to RFK or anyone high up in the FDA, please do. It is very safe, should have been approved 6 months ago. https://t.co/47ewUpt5K6

[Munchkin's Nana 🐈‍⬛👩🏻‍🦰🌊 @Munchkinsnana]

@VigilantFox Spasmodic Dysphonia can happen to anyone, it's not necessarily a vaccine injury and no scientific evidence shows that it's vaccine related. Anyone who is vaccinated could blame vaccines for any and every illness or condition they contract, that doesn't make it true.

[Karen P @catt30VA]

@dtr_trading SCORE

[Darp101 @DarpResearch]

Fantastic News from Larry Williams. Williams a NerveGen patient: his nerve pain has gone away!! https://t.co/jtRwqnO5Ke $NGENF Has a NVG-291 drug that allows spinal cord nerves to regenerate. He is an example of someone that has regained the ability to walk just after 6 weeks of treatment. The benefits keep coming, as once the nerves start to grow again, they keep going, even after the drug stops. Here is the most recent video interview of CEO https://t.co/IdStrw251G This drug should have been approved 6 months ago, superb safety profile and in FDA drug tests it has cured paralysis cases. If RFK knew, maybe he could push this thru.