Christina D

Please hear us, @FDA @FDA_KyleD.

For years, @hthurgood has advocated for Nathan before the FDA. How many times must families testify before their voices translate into meaningful action?

Patient engagement cannot become an exercise in documenting suffering without changing outcomes.

We have done our part. Our loved ones are running out of time.

#HuntingtonsDisease
#RareDisease
#CNPVforAMT130

Today, the @FDA granted Priority Review to @uniQure_NV AMT-130, bringing the first potential disease-modifying therapy for Huntington’s disease one step closer to patients.

I learned this news while my father lay in the ICU, on a ventilator, with aspiration-related pneumonia.

Pneumonia is the leading cause of death in Huntington’s disease. My father is now confronting one of the very complications that make this disease so devastating.

That is what the irreversible cost of waiting looks like.

A few years ago, he was still running the business he built over 30 years ago. An accomplished businessman, inventor, and published author. Today, Huntington’s disease has taken his independence, his ability to swallow, much of his ability to communicate, and now threatens his life.

My grandmother was diagnosed with HD in 1999. She died in 2011. Twenty-seven years after her diagnosis, we still have no FDA-approved treatment that slows this devastating disease.

As a little girl, I watched HD take my grandmother. Today, my daughter, named in her honor, is watching the same disease take her grandfather and great-aunt.

I am profoundly grateful for today's milestone. But I cannot ignore the human cost of getting here.

I welcome the FDA's decision to grant Priority Review to AMT-130 and strongly support its consideration under the Accelerated Approval pathway. Our community has contributed decades of natural-history research, patient-experience data, and evidence of our willingness to accept meaningful risk.

Every day of delay carries consequences that no subsequent approval can undo.

Years ago, my father asked me, “Christina, what are you doing to leave the world a better place?”

Dad, this is my answer.

I'm fighting for you, for our family, for the more than 41,000 Americans living with Huntington’s disease, and the 200,000+ at risk of inheriting it.

I'm fighting for a future where my daughter never has to stand before the FDA, pleading for a treatment to save her mother, her brother, her aunts, or another generation of our family.

I want her to inherit my grandmother's name.

Not her fate. And certainly not this fight.

💙💜

#HuntingtonsDisease
#RareDisease
#CureHD

@laurencurehd, I am incredibly proud of you. It takes extraordinary courage to stand before the FDA and make the most painful parts of your life public. Behind your words are the sacrifices, the weight of Huntington’s disease, and the people you love whose futures hang in the balance. You carried all of that with strength, grace, and conviction. Thank you for giving our community a voice when the stakes could not be higher.

💙💜

#HuntingtonsDisease
#RareDisease

I've waited for this day. After conversations with sickle cell advocates and outreach to @CMSinnovates, seeing plans to expand the Cell and Gene Therapy Access Model is deeply meaningful. For families facing Huntington's disease, scientific breakthroughs must translate into patient access. Thank you @DrOzCMS and @FDA_KyleD!

💙💜

#HuntingtonsDisease
#RareDisease
#GeneTherapy

An extraordinary story of scientific curiosity, innovation, and the power to change lives.

Professor Muramatsu helped transform decades of research into a life-changing therapy for a little girl.

Gene therapy is already changing lives in inherited blindness, sickle cell disease, and other devastating genetic disorders.

The @FDA now has an opportunity to demonstrate what leadership means for patients with devastating rare diseases.

The next generation of medical breakthroughs won’t be measured solely by what scientists discover, but by whether patients live long enough to benefit.

teachrare.org/from-frozen-ad…