AJ the Husky 🇺🇸

[AJ the Husky 🇺🇸 @AJ_Philly]

@HCLibertyLab @TracyBethHoeg @emilyakopp Nice try to deflect from the fact that same team rejected a drug for kids with Type 1 Diabetes single-handedly and the news came out two days ago how it unfolded.

[Lizzy Lawrence @LizzyLaw_]

The FDA has posted a memo that offers more insight into the disagreement between Høeg and the review team on this drug: https://t.co/aTGpWjatQR

[AJ the Husky 🇺🇸 @AJ_Philly]

@A_May_MD $ALMS and $VRDN are late stage. $IMVT also makes sense.

[AJ the Husky 🇺🇸 @AJ_Philly]

@anish_koka I don’t see any patient groups defending any of these clowns, if they were so great.

You’re the only useful idiot.

[AJ the Husky 🇺🇸 @AJ_Philly]

@anish_koka This you?

[Dogs&Viruses @Craigster771]

@TracyBethHoeg @anish_koka This is the type of person who defends you: reposts my post, blocks me so I can’t comment.

Zero class.

[AJ the Husky 🇺🇸 @AJ_Philly]

@Craigster771 @TrueTrueIke Or $VKTX for that matter.

[AJ the Husky 🇺🇸 @AJ_Philly]

@pawcio2009 Only one catholic slavic country on that list.

Kinda obvious

[AJ the Husky 🇺🇸 @AJ_Philly]

@HCLibertyLab @TracyBethHoeg You really need to ask yourself if you’re on the right side while you’re shilling for arrogant people who don’t care about patients and their families.

[Christina D @Christina4HD]

Katy, I appreciate your loyalty to Dr. Makary and I truly believe he wanted to reform a broken system. But as someone from the Huntington’s disease community, I need people to understand why so many families were deeply hurt by how we were characterized.

We were not “Big Pharma.” We were not paid lobbyists. We were families drowning in generational trauma because our loved ones are dying from one of the cruelest neurodegenerative diseases on earth.

And the hardest part? While many Huntington’s families, caregivers, and at-risk individuals were literally on Capitol Hill during Rare Disease Week meeting with lawmakers after paying our own way there, Dr. Makary was simultaneously speaking to reporters about “moneyed interests” and outside pressure surrounding AMT-130.

There were no corporate-funded advocacy campaigns behind us. Just families carrying decades of trauma, grief, and fear into congressional offices because we are terrified of running out of time. Nearly 50,000 signatures were gathered from HD families, grassroots advocates, caregivers, and supporters demanding urgency for AMT-130, and those petitions were hand-delivered directly to the FDA by members of the Huntington’s community themselves. Over 10,000 emails were also sent to FDA and members of Congress. Nobody paid us to do that. Fear did. Love did. Desperation did.

At the 2024 EL-PFDD meeting in Maryland, FDA sat across from more than 100 HD families and listened as we described watching parents, grandparents, siblings, and children slowly lose movement, speech, cognition, personality, and independence. People in those rooms cried with us. We were told urgency mattered.

Soon after we learned FDA aligned with uniQure on an accelerated pathway, many of us finally felt hope for the first time in our lives.

So yes, families became loud. Because this disease does not wait.

What also deeply damaged trust was watching Dr. Vinay Prasad speak anonymously to reporters about an active therapy. If someone cannot publicly face the people they serve during moments of crisis and scrutiny, they should not be leading CBER.

Leadership requires courage, transparency, and accountability. This is especially true in rare disease communities where every delay carries irreversible consequences.

Many of us respected Dr. Prasad intellectually. We respected his focus on scientific rigor. But from the patient/family side, much of what we experienced felt dismissive, detached, and at times unnecessarily combative toward a dying community simply asking to be heard.

And Dr. Makary protected that approach far too long.

The Huntington’s community is not asking for reckless science. FDA itself asked our community what level of risk we would accept, and nearly three-quarters of respondents said they would accept treatment risks for even a chance at 5 years without progression.

Because this disease does not wait for “perfect data.”

For families living with Huntington’s, every year means more neurons lost, more memories erased, more independence gone, and more people disappearing in front of us. AMT-130 showed the possibility of dramatically slowing that decline over three years. That is time with children. That is dignity. That is life itself.

We are not a lobbying machine. We are human beings trying to stop generations of suffering from continuing.

💙💜
#Huntingtonsdisease

[AJ the Husky 🇺🇸 @AJ_Philly]

@HCLibertyLab @DotknightWang @TracyBethHoeg Turned down life saving drugs
Created chaos, China is catching up to us
Ignored patient’s right to try
Shilled for influencers selling bogus supplements

On balance, a disaster group

[AJ the Husky 🇺🇸 @AJ_Philly]

@AzadehKhatibiMD @HCLibertyLab @TracyBethHoeg You expect her to tell everyone: I got fired because I take orders from Arnold Ventures, and I don’t put patients and unmet medical needs first?

[AJ the Husky 🇺🇸 @AJ_Philly]

@HCLibertyLab @TracyBethHoeg This clownshow turned down a drug for muscular distrophy. The drug cuts progression by half. Pure evil.

The company (Capricor) is anything but big pharma.

Please educate yourself.

https://t.co/Jq5Q1QGICK

[AJ the Husky 🇺🇸 @AJ_Philly]

@HCLibertyLab @TracyBethHoeg It’s amazing how little you know, you don’t even bother to inform yourself, and you’re stuck like a broken record with the same dumb catchphrases.

They. Don’t. Care. About. Patients.

That’s why they got fired.

[AJ the Husky 🇺🇸 @AJ_Philly]

@HCLibertyLab @RealJoeGrogan That reviewer would have been fired by RFKJr, the way he gutted all of FDA.

[AJ the Husky 🇺🇸 @AJ_Philly]

@HCLibertyLab An asset of Arnold Ventures. Never cared about patients.

Good riddance.

[AJ the Husky 🇺🇸 @AJ_Philly]

@HCLibertyLab @TracyBethHoeg Capricor has a drug that reduces progression in muscular distrophy and got rejected. Big mistake

She didn’t focus on patients and unmet medical needs

That is why she got fired. She ascribes to beliefs that severe patients aren’t worth the cost

This “big pharma” bs is tiresome

[AJ the Husky 🇺🇸 @AJ_Philly]

@TracyBethHoeg You didn’t focus on patients and unmet medical needs.

That is why you were fired.

[AJ the Husky 🇺🇸 @AJ_Philly]

@oakazaki The legal immigration process is a mess.

It seems this administration’s answer to everything is not to fix it but to just blow it up.

[AJ the Husky 🇺🇸 @AJ_Philly]

@CamillusMiura Did INCY fail on efficacy, or just preclinical safety?

[AJ the Husky 🇺🇸 @AJ_Philly]

@Arnold_Ventures @WSJopinion Your moral standards wouldn’t match even those of Hunter’s crack prostitutes

[AJ the Husky 🇺🇸 @AJ_Philly]

@EricTopol @Cell_Metabolism

[AJ the Husky 🇺🇸 @AJ_Philly]

@LuckyPenguin10 He’s a bought asset by Arnold Ventures.

As corrupt as it gets.