Marian Viñas

Abonada a lo insólito. Jefa de redacción en Non Stop People. Diletante en ECD.

[Marian Viñas @Marianvinas]

@OrvilleIV I will never surrender just like The #Coyote 🙃💪 Can we start a viral movement with powerful people, celebs, actors… to elevate 📣 awareness around #HD ?! 🪧🙏 all over the world?? @laurencurehd

[Ed. @VerEduard]

@laurencurehd @viatoCEO @HarrieScarlet @adamfeuerstein @DesertDweller93 @mike98572986 @peter_mantas @FDA The communication from Uniqure management was amateur at best. It is an open door for creating noise, mudding the waters and hiding the real positive signal of AMT-130 delaying the HD progression.
I am so sorry for the patients, this treatment is once more caught in drama.

[Lauren Holder @laurencurehd]

I am extremely disappointed by the way UniQure has handled this. Effective communication can make or break trials. This is a close-knit rare disease community. NONE of this would be possible without us. If we are showing support, if we are advocating for this, then we deserve support and respect in return. Effective communication is part of that.

[mike @mike98572986]

$qure $clpt

If I am sitting across the table from Matt, I am not spending 5 freaking minutes discussing rater bias and what might or might not have happened.

Time for Uniqure to get very serious and tell the stories of the actual patients. They can be anonymous for all anyone cares but stories win the day, not always numbers.

Over the years we have heard stories, the HD community wants to know what the patients are actually experiencing. You can talk about tfc all day until you are blue in the face, people want to hear how the patients started the trial and where are they now. How has amt 130 impacted their lives. The best pr they can do is just tell the stories of their patients. It isn’t very complicated. I am not sure why they are so tight lipped over this. I would also show plot charts. No reason they cannot do that.

If I had Matt’s ear, I would be harping on this all day everyday.

[Lauren Holder @laurencurehd]

I have done my absolute best to be honest and transparent with everyone. I genuinely believe in this treatment - a big part of that is because I have seen that it works with my own eyes. Lived experience goes a long way when it comes to being confident something is working, and to personally know some of the participants and know how they are doing now vs before - for me, I have no doubt to the efficacy of the high dose.

[DesertDweller_ROAR_4_A_CURE_4_HD @DesertDweller93]

I am just an investor in $QURE while those like @laurencurehd are living with HD. My frustration with how the update was communicated is not just related to my investment, I understand there are real people who need this treatment.

I really wish @uniQure_NV would have done a better job with messaging about how great it is working. Too much uncertainty with how the story got out and was communicated. Come on management, do better, don't let the community down.

The confusion surrounding the "Missingness" of the patients in the natural history arm was the first time it has been brought up. This should have been explained earlier to avoid the confusion. The community has already suffered enough with all the drama earlier this year, don't add to that.

[Master Sea🌊 @Master_SeaSeo]

Both Walid & Dr. Sung confirmed that "one outlier pulled the average down significantly." @uniQure must proactively release individual trajectories & outlier-excluded sensitivity data.

Transparency is what shareholders and HD families deserve, Matt Kapusta. $QURE

[Migue @sinbuenostuits]

*le pregunto algo a ChatGPT*

ChatGPT:

[Onvre Deconstruido @o_deconstruido]

Antológico 😂🤣👇🏻

[Christina D @Christina4HD]

@FDA @FDADrugs I have watched three members of my family suffer from Huntington’s disease, and its shadow still hangs over us.

HD has no approved treatment that slows or stops its progression. No one in my family has ever had access to a clinical trial.

Families are watching the people they love slowly die from a devastating disease while caregiving, working, raising families, and fighting through a system that can move far too slowly.

We hear often about the need for urgency in rare disease. We need to see that urgency translated into action.

Give families a meaningful chance to access promising therapies and clinical trials. For people facing relentlessly progressive, fatal diseases, the risk-benefit calculation is fundamentally different.

Why should families already carrying an unimaginable burden have to fight so hard for a chance?

We need a regulatory system that can move faster than the diseases taking our loved ones from us.

[Mads Lindegaard Rosendal @MadsLRosendal]

@peter_mantas @johnkmason @ripper1399 @mike98572986 @DesertDweller93 @HYcorps Why haven’t we heard from EMA on AMT 130 ? They’re awaiting FDA approval as usual ?

[Archaeo - Histories @archeohistories]

She never called her family to say goodbye. Instead, Sara Low used her final 30 minutes to make sure the FBI knew who attacked America.

Trapped at 30,000 feet with no weapon, 28-year-old flight attendant Sara Low held the only tool that could expose the 9/11 hijackers to the world: 14 numbers memorized in her head.

When terrorists stormed the cockpit of American Airlines Flight 11, chaos erupted in the cabin. Flight attendant Amy Sweeney scrambled to grab an onboard airphone to alert the ground team, but hit a devastating wall - she didn't have a calling card to bypass the system lock.

Without her wallet on hand, Sara stepped forward. She didn't have plastic, but she had clarity. She had memorized her family’s AT&T calling card digits.

Sara handed those numbers to Amy, unlocking a lifeline to the ground.

For the next 20 minutes, Amy stayed on the line with American Airlines flight services in Boston. Because of Sara's calling card, the FBI received its very first concrete intelligence of the day: exact seat numbers of the hijackers, physical descriptions, and their movements in the cabin.

While Amy relayed the critical data, Sara spent her final half-hour moving terrified passengers toward the rear of the plane, shielding them from danger.

Sara had the means to use that airphone. She could have called her parents. She could have said a final goodbye to her sister.

She didn't. She chose to make sure the world would know the truth instead.

True heroism isn't defined by muscle or weapons. It’s defined by presence of mind in the face of impossible terror - and the selfless decision of who that mind chooses to serve.

#archaeohistories

[Master Sea🌊 @Master_SeaSeo]

THIS is the kind of lawmaker people actually need.

Meeting patients and frontline clinicians at the Huntington's center to cut bureaucratic red tape and push for faster access to life-changing therapies. Leadership that truly listens. 🏛️👏 $QURE

[Dr. MoneyBall @Dr_Moneyball]

Dr. Sung on the $QURE Guggenheim KOL call just now:
He wouldn't be surprised if the 4-year data shows a 85-90% slowing of cUHDRS if AMT-130 patients remain relatively stable.

[DesertDweller_ROAR_4_A_CURE_4_HD @DesertDweller93]

@uniQure_NV $QURE management, please release the 4-year data this week, ahead of this important meeting so AMT130 can be THE topic of this important meeting.

[DesertDweller_ROAR_4_A_CURE_4_HD @DesertDweller93]

Open plea to @uniQure_NV $QURE management. Please release the 4-year data before next week so if Dr Sung speaks on 9/8/26 at Guggenheim conference and with @Help4HDI conference on 9/11 & 9/12 they can talk about new data and not 1 year old data.

Thanks for your consideration.

[Wall Street Wolverine @wallstwolverine]

Javier Bardem sobre la crisis de Ceuta: “Lo que ha sucedido es una forma de manipulación de Marruecos frente al Gobierno. Arrollidarnos frente a Marruecos es saber que pueden hacer lo que quieran con el grifo de la inmigración”.

[Miguel Ángel Bernal Sánchez-Roxado @mabernalsanchez]

PEDRO SÁNCHEZ HA CONSEGUIDO QUE SUENE EL HIMNO DE ESPAÑA EN BILBAO.

[Lauren Holder @laurencurehd]

Let me also clarify that Novartis is enrolling for their trial right now and participants will be blinded for 36 months. Here are the differences to me (as a person actually able to participate in these trials):

-Novartis' doesn't have 4+ years worth of data and people with lived experience already proving the treatment works.
-I have been very honest that I feel, even in the case of the Novartis trial, that a 36-month placebo arm in HD is unethical. TIME IS BRAIN. Irreversible damage is happening, progression is occurring and can't be reversed.
-Novartis treatment is a pill with a washout period. This means that if something incredible appeared in those 36 months, I could stop participating, do the washout period, and still participate in other trials.