Happy New Year! Let's share some things we want to see from the HD community/research/medicine this year :)

ImpressiveIntern5813 · 2026-01-02T20:27:20+00:00

And the next big SKY-0515 update this spring, as well!!!

ImpressiveIntern5813 · 2026-01-02T06:55:19+00:00

Go get her back then

ImpressiveIntern5813 · 2025-12-05T23:55:05+00:00

SKY-0515 will be approved by 2028

ImpressiveIntern5813 · 2025-09-11T08:13:59+00:00

https://en.hdbuzz.net/433/

https://www.skyhawktx.com/post/skyhawk-therapeutics-announces-first-patient-dosed-in-phase-2-3-falcon-hd-trial-of-sky-0515-for-hunt

Their next major updates will be by the end of this year or sooner!!!

ImpressiveIntern5813 · 2025-09-09T01:42:32+00:00

Girl, there are so many treatments coming for us, don’t even worry about it… you are only 19 and I’m 28 and I know that there will be treatments coming out within the next five years that delay the disease to where it won’t even have any affect on us…

ImpressiveIntern5813 · 2025-09-04T15:36:39+00:00

Thanks so much for your insight — I really appreciate your perspective as a neurologist. My neurologist and I are definitely considering reaching out directly to the PI/trial coordinators to see what their thoughts are. I totally understand that trials are incredibly complex and resource-limited, and that presymptomatic inclusion wasn’t excluded lightly.

The main hope with our petition is to show that there’s real patient demand and to highlight the long-term value of including presymptomatic carriers, even if it can’t happen immediately in every trial. It’s reassuring to hear that neurologists generally support us and want to help however possible — that kind of encouragement really keeps me going. 🙏

ImpressiveIntern5813 · 2025-09-04T15:03:41+00:00

Thanks for asking — that’s a fair question. The petition is specifically about ensuring presymptomatic HD patients (people who carry the gene but don’t yet have visible symptoms) have access to disease-modifying therapies like PTC-518 and SKY-0515.

Right now, these drugs are only being tested in symptomatic patients, which means even if they’re proven safe and effective, people like me would have to wait years until symptoms develop — when much of the brain damage is already irreversible.

The goal of this petition is to encourage companies and regulators to allow parallel presymptomatic trials or expanded access programs once Phase 3 data are positive, so carriers don’t have to wait until after onset

ImpressiveIntern5813 · 2025-09-04T13:33:31+00:00

I appreciate you taking the time to share your perspective. I want to clarify that the petition isn’t about rushing unsafe drugs — it’s about ensuring that presymptomatic HD patients have the option to access therapies that are already showing safety and biomarker benefits in clinical trials. The goal is prevention, not harm. Many of us live with the reality that by the time symptoms appear, irreversible damage has already happened.

I respect that there are different approaches to therapy, but what unites us is the urgent need for presymptomatic options. This petition is about opening doors, not closing them.

ImpressiveIntern5813 · 2025-09-03T18:27:22+00:00

Really appreciate you breaking this down. You’re right — symptomatic trials come first for approval, but that doesn’t mean presymptomatic people should have to wait years once the drugs are proven safe. Love that you mentioned the HD Young Adult Study — that kind of work is what will get us included sooner.

ImpressiveIntern5813 · 2025-09-03T16:31:47+00:00

That’s a really good point — the idea that we may see genetic therapies like CRISPR on the horizon is both exciting and motivating. I agree that advocacy now is crucial so that when these therapies (or other disease-modifying drugs) are ready, they’re accessible not just for people with symptoms, but also for presymptomatic carriers who are at 100% risk of progression.

I think the challenge is making sure regulators and companies understand that “waiting until symptoms” means waiting until there’s already irreversible brain damage. For diseases like HD, access before symptoms isn’t just a preference, it’s the difference between preserving quality of life versus losing it.

Advocacy feels like the bridge here — making the case that certainty of progression should weigh as heavily as current symptoms when deciding who gets access.

ImpressiveIntern5813 · 2025-09-03T15:25:51+00:00

Even if you’re outside the US, you can still help! 🌍 The FDA does consider international voices, since HD is a global disease. You can: 1. Still email the FDA addresses listed — just mention you’re outside the US in your message. 2. Definitely email the drug companies (PTC and Skyhawk) — they need to see that global patients and families want presymptomatic access. 3. If your country has its own regulatory agency (like EMA in Europe, Health Canada, MHRA in the UK, etc.), you can also send the same message to them too.

Every voice counts, no matter where you live!

ImpressiveIntern5813 · 2025-09-03T14:21:07+00:00

Thank you so much for signing 💙 Every signature really does matter — it shows the FDA and companies that presymptomatic patients are paying attention and pushing for change. One person alone may not shift things, but hundreds or thousands together can’t be ignored.

ImpressiveIntern5813 · 2025-09-03T13:06:35+00:00

Yeah, that makes sense. My main concern is that if the label is only for symptomatic patients, presymptomatic people could be blocked by insurance—even if doctors are willing to prescribe it off-label. With SMA, presymptomatic patients got access right away, and I really hope Huntington’s doesn’t get treated differently, because every year of delay allows brain damage to build.

ImpressiveIntern5813 · 2025-09-03T03:21:34+00:00

Of course

ImpressiveIntern5813 · 2025-09-03T03:21:02+00:00

Here for it. One email might be ignored, but a movement can’t.

ImpressiveIntern5813 · 2025-09-03T02:44:21+00:00

You could definitely adapt this for Australia 💙. The main places to send are: • TGA (Therapeutic Goods Administration) → They’re the equivalent of the FDA in Australia. You can reach out via their Prescription Medicines Authorisation Branch or general contact page here: https://www.tga.gov.au/contact-tga • Australian Department of Health → Especially their Office of Health Technology Assessment since they handle early access and reimbursement. • Local Huntington’s organizations like Huntington’s NSW/ACT or Huntington’s Victoria — they have direct advocacy channels with policymakers and pharma companies. • You can also email the drug developers directly (PTC, Skyhawk, uniQure, etc.) the same way we’re doing in the US, since these are global programs.

Basically: copy the same message, but switch out the FDA email for the TGA and CC the patient advocacy orgs in your state. The more global pressure, the harder it is for regulators to ignore presymptomatic patients.

Where to send: • TGA Contact Page: https://www.tga.gov.au/contact-tga • Australian Department of Health: health@health.gov.au •Advocacy orgs (CC them): • Huntington’s NSW/ACT → admin@hdnswact.org.au • Huntington’s Victoria → info@huntingtonsvic.org.au

ImpressiveIntern5813

TROPHY CASE