Allison Kennedy

Huntington’s Disease Advocate 💙 hypermobile Ehlers-Danlos Syndrome Advocate 🦓

[Dr. Toonces, MD, PhD @DrToonces]

Hard to picture a better year for uniQure to be at the HD community's convention.

FDA's aligned on the 3-year data. BLA set for Q3. The momentum is real.

#AMT130 #HDSAConvention

[Christina D @Christina4HD]

“The uniQure decision ultimately represents what every rare disease advocacy group sets out to do - develop a promising treatment, put it before regulators, and offer a chance to alter the lives of the rare disease patients they represent. And while that may be the final step in the process -- to get there hundreds of things need to go right.”

Well said, @bquick83. As a Huntington’s disease family member, I agree. Rare disease families understand how much has to go right to reach regulators, and how meaningful these moments are for those waiting.

[uniQure @uniQure_NV]

Today we provided a regulatory update on a path forward for AMT-130 for #huntingtonsdisease. Read the full update: https://t.co/ShR79YQMq5

[Dr. Toonces, MD, PhD @DrToonces]

Huntington's disease in numbers:

30,000 Americans currently symptomatic
200,000+ Americans carry the gene
0 disease-modifying treatments approved
1 drug with 75% slowing of progression in trials
1 FDA reversal blocking access

This is what a regulatory failure looks like in human terms.

$QURE #AMT130

[CP2 @LuckyPenguin10]

Yes, this is the question many of us have.

In your last press conference on rare diseases, you alluded to several therapies the FDA had effectively blacklisted.

With everything that's come out since, it's increasingly clear that $QURE AMT-130 was unethically stonewalled by former CBER Director Vinay Prasad.

> The data is strong.
> The patients want and need it.
> The leading HD experts have come out in support it.

@SenRonJohnson – can you please provide an update to those investigative efforts into the @US_FDA?

The HD community is losing time.

—
@laurencurehd
@rachelreising96
@Christina4HD
@DanielleValenti
@afkennedy8

[Allison Kennedy @afkennedy8]

@AndrewNixonHHS @mike98572986 @DrMakaryFDA My mom is 1 of 41,000 Americans impacted by Huntingtons disease every day. I am one of over 200,000 + at risk for having the HD gene. It’s so nice to know that HD is so high on your priority list. DO BETTER! This is UNACCEPTABLE! #IAmTheSwamp #Huntington’sDisease