Laura Ford

[Lauren Holder @laurencurehd]

I’m glad you put a “bias alert” at the beginning, because at least that part is honest:

“I’m on a side. I’m not impartial or fair. I’m biased with a punch-anyone-in-the-face-who-talks-shit-about-my-family kind of bias.”

The problem is that this bias seems to extend to dismissing rare disease patients and advocates before ever actually listening to them.

When you reduce rare disease patient groups to people simply being “told by Pharma” that the FDA is delaying some “wonder drug,” that’s not thoughtful analysis. That’s caricature. And it’s insulting.

Patients in communities like Huntington’s disease are not naïve props being manipulated into outrage. Many of us have spent YEARS - some of us decades - immersed in this work. We’ve participated in clinical trials, sat in FDA meetings, and helped shape patient-focused drug development. We've learned the science because our lives and families depend on understanding it.

You do not have to agree with every patient advocate. You can absolutely question data, regulatory decisions, company claims, or trial design.

However, dismissing patients as if we are incapable of independent thought because our perspective doesn’t align with yours is arrogance, not objectivity.

And frankly, the assumption that desperate patients are too emotional, too compromised, or too uninformed to think critically for themselves says far more about the person making that assumption than the patients being dismissed.

Yes, patients are emotional. Of course we are! We are watching people we love decline in real time. Some of us like myself are living this in our own bodies. We understand exactly what lost time means.

Emotion does NOT negate intelligence. It does NOT erase lived expertise. And it certainly does not mean patients are incapable of understanding risk-benefit tradeoffs in diseases that are relentlessly progressive and ultimately fatal.

What’s actually dangerous is a worldview that treats patient voices as disposable the moment they become inconvenient.

If your starting assumption is that rare disease communities are simply being manipulated rather than engaging thoughtfully with extraordinarily complex issues, then you (and @DrMakaryFDA and @VPrasadMDMPH) were never actually trying to hear us in the first place.

[Help4HD @Help4HDI]

Inspired by the advocates, families, and community members, five Huntington’s disease organizations came together today to champion the future of rare disease innovation at the FDA.

Together, we look forward to strengthening our partnership with the FDA to bring renewed hope, urgency, and patient-centered progress to Huntington’s disease.

Every day matters for HD families — and we will continue advocating together for a brighter future. 💙

#HuntingtonsDisease #RareDisease #PatientAdvocacy #HDCommunity @POTUS @realDonaldTrump @WhiteHouse @US_FDA @FDACBER @FDACDERDirector @RFKJr_Official

[Rachel Reising @rachelreising96]

Signed by myself and a couple others in the rare disease community💙 honored to represent multiple rare diseases @POTUS @realDonaldTrump @RFKJr_Official

https://t.co/pXMOu1m2Ml

[Houman David Hemmati, MD, PhD @houmanhemmati]

The FDA Commissioner role is open. This is a pivotal moment for American medicine — & for American patients.

Here’s what I believe the next Commissioner should stand for. Not as a wish list. As a baseline.

🔬 1. BRING BACK THE ADCOMS — AND MAKE THEM COUNT.
Advisory Committee meetings are one of the FDA’s most powerful tools for transparency. They should return in full force. Open. Public. And real.

Patients, doctors, scientists, advocates, & skeptics should all be able to speak. But if you want to speak, you fill out a financial conflict-of-interest form under penalty of perjury — & you read it out loud at the podium before you say another word. Every single person.

And if the issues are complex and the science requires more than one day, then take the time. Don’t cut people off because the schedule says so. The public deserves to see exactly how these decisions are made. Real transparency builds real trust.

⚖️ 2. STOP ASKING ONLY “IS IT SAFE ENOUGH TO APPROVE?” — START ASKING “WHAT HAPPENS IF WE DON’T?”
For rare diseases. For serious conditions with no good options. For patients who don’t have ten years to wait for traditional trials that may never be feasible.

Every regulatory decision carries two risks: the risk of approving something, and the risk of not approving it. Both are real. Both affect real people.

When traditional gold-standard trials aren’t practical, we should still be able to move with urgency — but only when paired with strong post-approval commitments and rigorous safety monitoring. We can give desperate patients a chance without abandoning scientific integrity.

🇺🇸 3. THE FDA’S ONLY CLIENT IS THE AMERICAN PATIENT. FULL STOP.
The FDA exists to serve patients — not outside interests or external pressures of any kind.

It must continue protecting the public from products that carry real, known risks but offer no meaningful clinical benefit.

At the same time, when there is credible evidence that a treatment can help patients with serious conditions, Americans and their physicians should be trusted to make informed decisions once they have complete and honest information about the risks, benefits, and alternatives.

The FDA’s job is to make sure the science is sound and the information is clear. Then let patients and doctors do what’s best for them.

These principles matter because the FDA’s decisions affect every family in this country.

Note: This is a simple social media post and not a massive policy document. The issues are FAR more nuanced, there are challenges and risks with each of the things I said above, and I recognize that. But it's intended to serve as a discussion starter. We must always strive to improve, and we can when we have open debate and dialogue.

[Christina D @Christina4HD]

AMT-130 represents the most promising disease-modifying approaches currently being studied in Huntington’s disease, aiming to target the underlying genetic driver rather than just symptoms. In Huntington’s disease, where there are no approved treatments that slow progression and time is running out for patients, regulators should allow more flexibility and act with urgency. The goal isn’t to lower standards, but to keep the pace of review aligned with the pace of the disease.

That’s why it’s important to be precise with the data. Small exploratory cohorts (like n=17) and early 12-month readouts are not designed to determine disease acceleration or efficacy in Huntington’s. HD is slow, variable, and requires longer observation windows to interpret true treatment effects.

This is also why the 3-year follow-up data is so important. Long-term trends rather than early short-term fluctuations are what shows AMT-130 produces durable slowing of neurodegeneration.

What often gets lost in these debates is the human reality behind the science.

Over 50,000 people in the Huntington’s disease community have signed petitions supporting faster access for therapies like AMT-130. This is not driven by “big pharma,” but by patients, families, caregivers, and volunteers living with a relentlessly progressive disease and limited options. There is no financial incentive, only urgency.

In 2024, many of us met with the FDA in Maryland through the HDSA EL-PFDD meeting. I personally shared my story and saw firsthand how deeply these experiences land with regulators. We were told patient voices mattered and that accelerating pathways for serious neurodegenerative disease was a priority.

In the 2024 HDSA survey, 3/4 of respondents would accept meaningful risk for 5 years without progression, and ~35% would accept risk even for 3 years of halted disease.

We are not “moneyed interests.” We are patients and families asking for decisions grounded in full, long-term evidence, and for regulatory flexibility and urgency that match the pace and severity of this disease.

[Laura Ford @LauraFord]

Amazing @Help4HDI #HIPE event in Baltimore over the weekend. It was my husband’s first HD ed event. He’s not a fan of crowds or sitting or listening. I know he went because he loves me, he loves our family, and he sees this community loving him on his HD journey. 💙🩵

[Lauren Holder @laurencurehd]

I didn't get the chance to say what I wanted earlier because my kids needed my attention and I've been packing and getting ready to leave for the @Help4HDI #HIPE event in Baltimore this weekend. But this article really got me worked up.

I appreciate the acknowledgment from Dr. Buracchio that the “plausible mechanism framework” could extend beyond individualized therapies and shouldn’t disadvantage diseases like #HuntingtonsDisease.
But the reality is, we are ALREADY seeing a double standard.

The FDA is signaling openness to flexible, mechanism-based approaches while rejecting those same principles when applied to Huntington’s and AMT-130.

That’s not just confusing;
It’s UNACCEPTABLE for a rare disease community that depends on regulatory consistency.

The comparison to “Baby KJ” highlights a fundamental disconnect:
In pediatric conditions, success is defined by improvement - meeting milestones, gaining abilities.

Huntington’s does not work that way. This is a progressive neurodegenerative disease where the most meaningful outcome is slowing decline or maintaining function longer than expected.

In HD, stability IS improvement.

So when the FDA says it needs a “clear and distinct” effect from natural history, the question is:
What does that look like in a disease where decline is the baseline?

Because if converging evidence across clinical measures like cUHDRS, biomarkers like NfL, and natural history comparisons isn’t enough in HD, then the standard isn’t just high - It’s completely misaligned with the biology of the disease.
And when standards are misaligned, patients pay the price.

This isn’t about asking for exceptions. It’s about expecting the @US_FDA to apply its own stated principles consistently and in a way that reflects the realities of the diseases it regulates.

Because in Huntington’s disease, delay isn’t neutral.
Delay is decline.
Decline is irreversible.
And irreversible means time we don’t get back. #TimeMatters #DelayStealsTime #ActWithUrgency
@adamfeuerstein @SenateAging @SenRonJohnson @SenRickScott @SenGillibrand @RepAuchincloss @FDACBER @DrMakaryFDA @realDonaldTrump @WhiteHouse @fdacder @FDACDERDirector @houmanhemmati

[Laura Ford @LauraFord]

“Justice and equity don't come from telling troubled communities what they need to do, but from listening to those having trouble and working together to forge solutions.…”

DOE Seeks Public Input on Environmental Justice Strategic Plan https://t.co/ZJqJWK0afI

[Laura Ford @LauraFord]

I’m thrilled to be starting a new adventure as Grants Program Specialist at @foreversolar. Can't wait to dive into new opportunities to forge partnerships for people and the planet. #SolarIndustry #ConstantlyGrowing #ShakingThingsUp #GoodEnergy

[Laura Ford @LauraFord]

After an incredible 20-year journey as part of the @Accokeek staff, I've transitioned to a new chapter, now caring as a volunteer for @PiscatawayPark Indigenous cultural landscape and its rich stories. I’ll be forever grateful for the lessons learned on this landscape!

[Laura Ford @LauraFord]

Holy Week - The Atlantic. “Fifty-five years ago today, Martin Luther King Jr. was assassinated. In stories about the civil-rights movement, what happened next is often overlooked. But the week that followed his killing changed the course of history….” https://t.co/1t9NqkVRsT

[National Park Service @NatlParkService]

To avoid crowds, visit areas that are less crowded.

[Laura Ford @LauraFord]

Grateful for this grant from @NationalParkFdn and honored to be part of the team @Accokeek working to expand interpretation by Native People, for Native People @PiscatawayPark.

[National Park Foundation @NationalParkFdn]

Philanthropy and partnerships are essential to the success of America’s national parks. NPF is committing more than $1.7 million to cultivate and expand national park philanthropy through the Strong Parks, Strong Communities program. https://t.co/ZlYwAqgqvz

[Laura Ford @LauraFord]

Artists in Business Leadership — First Peoples Fund https://t.co/4wcs5CPPHi

[Laura Ford @LauraFord]

NDN Collective Launches 2022 Community Self-Determination Grant: A Power Building Opportunity for Indigenous Communities https://t.co/BF2OMovhjY

[Laura Ford @LauraFord]

https://t.co/EvPsd1ZZt2

[Fairfield Foundation @fairfieldfound]

Internship opportunity!!! This is for tribal members aged 18-30 to assist with the planning and protection of the sacred native site of Werowocomoco in Yorktown, VA, as well as gain experience working with the National Park Service.

[Laura Ford @LauraFord]

Restoration depends on us recognizing the stewardship and knowledge of Indigenous people and honoring the sacred connections between land and people. #earthday @accokeek ⁦@PiscatawayPark⁩ https://t.co/w86sd8IY0m

[Laura Ford @LauraFord]

"By recognizing the significance of certain locations we provide an opportunity for people of all backgrounds to gather and acknowledge both the painful and triumphant moments that occurred there."
Protecting Our Roots by @USFWS https://t.co/ZwR0INNnKN

[Laura Ford @LauraFord]

“Traditional models of protected area management… are simply not adequate to address the needs of a world confronting climate change, urbanization, and the loss of biodiversity – as well as long overdue reckonings on racial justice and colonialism.” https://t.co/R0vWb18MfP