Dr. Jan Nolta

California Institute for Regenerative Medicine @CIRMnews · Sep 25

“Every person in CA should be able to access the most promising medical advances, including cell & gene therapies that are changing what is possible for serious and life-threatening diseases.” - Shyam Patel, Associate VP of Patient Access. Press release: bit.ly/4hfsdkg

View quoted post on X

Lauren Holder Verified account @laurencurehd · Jun 17

Today feels like a victory for the entire #HuntingtonsDisease disease community.

After months of uncertainty, difficult conversations, and tireless advocacy, we received the news that uniQure will be able to move forward as originally planned.

For many of us, like myself, this is deeply personal. We know what it means to watch the clock in HD. We know what it means to live with the reality that time lost cannot be recovered.
That's why this moment matters so much.

It's also important to acknowledge that this happened because people spoke up - patients, families, caregivers, researchers, clinicians, and advocates came together and REFUSED to let our voices go unheard.

We shared our stories. We signed petitions. We met with lawmakers and regulators. We educated others about what delays mean in a progressive, fatal disease where every day matters.

Today is a reminder that patient voices matter. Lived experience matters. Advocacy matters.

To everyone who signed, shared, spoke out, listened, supported, and stood beside this community: THANK YOU.

Let's take a moment to celebrate what we accomplished together.
Today, it feels like we were finally heard.
#TimeMatters #StandUpSpeakUp4HD #DelayIsDamage #RareDisease
@Help4HDI @FDA_KyleD @US_FDA @HDYOFeed @hdfcures @HDBuzzFeed @FDACBER @RepAuchincloss
@houmanhemmati
@JRenz0418
@Christina4HD
@rachelreising96
@BrittanyMegan10
@POTUS @senronjohnson @flsenrickscott

View quoted post on X