A plain-English, source-verified view of where HD research actually stands — advances and setbacks given equal weight.
Version 2.8 · Last checked October 12, 2026 · Last substantive change September 29, 2026
This is information, not medical advice. Nothing here should guide a treatment or trial decision on its own. Those conversations belong with your neurologist or HD care team — and HDSA (1-800-345-HDSA) can help you find an HD specialty clinic.
Every factual claim on this page is dated and linked to its source — ClinicalTrials.gov, peer-reviewed journals, HDBuzz, HDSA, CHDI, or a company's own press release. Where a claim couldn't be fully verified, it says so. The pipeline moves fast; the two dates above tell you how fresh this snapshot is — "last checked" is the last full sweep of the field, and "last substantive change" is the last time a fact on this page actually moved.
Three rules keep this honest:
Status labels used in the tables:
"Recruiting" means the trial is enrolling new participants as of the verification date. "Active, not recruiting" means it's running but closed to new participants. Discontinued programs stay on this page permanently — knowing what failed, and why, matters as much as knowing what's next.
September 2025 through September 2026 was arguably the most consequential year HD research has ever had — in both directions. One therapy became the first aimed at slowing HD to reach an FDA application — a submission, not an acceptance or approval — even as its latest four-year data landed short of the hopes attached to it; another, after fifteen years and roughly 1,500 participating families, was abandoned. Advances and setbacks below, ordered by weight rather than by good news — use the controls to filter or re-sort by date.
On Sep 29, 2026, uniQure released 48-month data on the 12 high-dose patients. Four years after the one-time brain surgery they were still estimated to be declining more slowly than a matched comparison group of untreated patients — but the combined clinical scale (cUHDRS), the headline measure, no longer reached statistical significance (44% slowing, p=0.144). The daily-function score (TFC) did stay statistically significant (61% slower decline, p=0.008). A separate comparison against an older external-control dataset looked stronger (cUHDRS 53.5% slowing, p=0.041), but swapping comparison groups after the fact is weaker evidence, not stronger — and as HDBuzz noted (Sep 30), the result swings on which control data you use. uniQure's shares fell about 40% on the news (more steeply intraday). On safety, five patients — about 17% of everyone who received the high dose, a larger group than the 12 who reached four years — had treatment-related serious brain inflammation, all reported resolved; a suicide in a low-dose patient about five years after treatment was assessed as unrelated. What this does and doesn't change: the FDA application still rests on the earlier, stronger 36-month data (80% slowing on the combined cUHDRS score vs. an external comparison group, p=0.005); the BLA (submitted Sep 2, 2026, with a parallel UK MHRA filing) is still only submitted — not accepted, not approved. It is still the only effort to slow HD to reach an FDA application, but the four-year readout is a real check on how strong that signal really is, not the confirmation many hoped for.
On Jul 9, 2026, Roche announced that in GENERATION HD2, tominersen (a spinal-injection ASO) lowered huntingtin and improved biomarkers but did not slow clinical decline at 16 months. The program — HD's largest ever — is discontinued, along with the next-generation ASO RG6496 (stopped over animal-toxicology findings after just three participants were dosed). Roche's letter to the HD community. Roche's gene therapy (SPK-10001, below) continues unchanged.
Novartis's INVEST-HD Phase 3 (NCT07326709) began Mar 24, 2026 and is recruiting ~770 participants at sites across the US (see the trial-finder section). Supporting it: Apr 28, 2026 data showing dose-dependent slowing at 24 months versus natural-history comparisons. Caveat: that 24-month data is open-label against external controls; the real Phase 3 answer is roughly three years away.
On Nov 3, 2025 the FDA unexpectedly rejected the accelerated-approval path it had earlier signaled, and by Mar 2, 2026 was recommending a whole new randomized trial — before reversing again in June. The episode cost eight months and shows how unsettled the regulatory ground still is for HD therapies built on small, externally-controlled datasets.
Skyhawk's oral pill lowers both huntingtin and PMS1 (a DNA-repair gene tied to disease progression). Its final 15-month Phase 1/2 results (Sep 15, 2026) showed ~60% lowering of mutant huntingtin and ~25% reduction in PMS1 at the high dose, no serious safety events, and a combined clinical score (cUHDRS) that rose slightly on drug (+0.94) while an external comparison group declined (−0.65) — a difference reported as statistically significant (HDBuzz). The pivotal FALCON-HD trial expanded its enrollment target from 400 to 600. Strong caveat, unchanged: this is 15 people in an open-label study with no placebo — the clinical-score signal rests on a comparison to historical data, not a randomized control. Encouraging that it held, but the blinded FALCON-HD readout (2027 at the earliest) remains the real test.
After a negative opinion from the EMA's expert committee in July 2025, Prilenia withdrew its EU marketing application on Nov 7, 2025. The Phase 3 PROOF-HD trial had missed its primary endpoint (published in Nature Medicine, Sep 5, 2025). A new confirmatory Phase 3, PRECISE-HD, began Jul 7, 2026 — testing the drug only in the subgroup where a post-hoc signal appeared. A legitimate but long-odds retry; first answers ~2028.
Sangamo Therapeutics — whose preclinical zinc-finger repressor was the leading epigenetic approach to silencing mutant huntingtin — filed for Chapter 11 in June 2026. Eli Lilly bought the zinc-finger platform (Aug 12, 2026), but no buyer has said it will develop the HD candidate. Its status is genuinely unknown.
The newest strategy in HD — blocking the DNA-repair errors that let the CAG mutation grow over a lifetime — produced its first wave of drug candidates this year: LoQus23's oral MSH3 inhibitor LQT-23 (Jan 6, 2026), Rgenta's oral PMS1-lowering RGT-0474060 (Feb 25, 2026), and Latus Bio's MSH3 gene therapy backed by a $97M raise (May 4, 2026). Perspective: every one of these is preclinical. Not a single dedicated somatic-instability drug has been given to a human yet. First trials are realistically 2027.
Sarepta's SRP-1005 became the first huntingtin-lowering therapy given by subcutaneous injection — no spinal tap, no brain surgery — when the Phase 1 INSIGHTT trial began dosing on Aug 20, 2026 (New Zealand only so far). Alnylam/Regeneron's spinal-injection siRNA ALN-HTT02 is also recruiting its Phase 1 (NCT06585449; no US sites yet). Both are safety-stage — years from any efficacy answer.
Vico's CAG-targeting ASO showed encouraging protein-lowering in a tiny group (three patients), but several participants developed radiculitis (nerve-root inflammation). Vico switched to twice-yearly dosing (Feb 24, 2026), partly to address this, with US sites planned for late 2026. Worth watching the safety data as closely as the efficacy data.
The dates below are expected, not promised — drug timelines slip, and an anticipated readout can be delayed, missed, or come out mixed. This is here so you know what's on the horizon and when it's worth checking back, not as a forecast of good news. Ordered by how soon each could land.
Moving faster than this page? This tracker updates on a schedule, so a development can break before it appears here. For the latest between updates, check HDBuzz (plain-language HD research news) and HDSA, or — for AMT-130's FDA progress specifically — uniQure's news releases. Expected timelines above are compiled from company guidance, ClinicalTrials.gov, and HDBuzz as of the "last checked" date; when any of these actually happens, it moves up into "What's moved."
Grouped by how each therapy works. Within each group, active programs come first, then discontinued ones — kept visible on purpose. "Latest readout" gives the most recent result and its date; read it alongside the caveats above. All statuses verified Oct 12, 2026; where a public trial registry lags a company announcement, that's noted.
Synthetic molecules delivered by spinal tap — or, newly, under the skin — that tell cells to make less huntingtin protein. Must be re-dosed. The most-tested strategy in HD, and the source of its biggest failure.
| Program | Mechanism | Phase | Trial | Status | Latest readout |
|---|---|---|---|---|---|
| ALN-HTT02Alnylam / Regeneron | siRNA, spinal injection; silences huntingtin (targets exon 1) | Phase 1 | NCT06585449 | Recruiting No US sites yet (UK, Canada, Germany) |
No human efficacy data yet. Design presented at CHDI Feb 2026. Registry |
| SRP-1005 (ARO-HTT)Sarepta | siRNA, under-the-skin injection — no spinal tap or surgery | Phase 1 | NCT07536061 | Recruiting New Zealand only so far |
First-in-human dosing began Aug 20, 2026. Safety-stage. HDBuzz |
| WVE-003Wave Life Sciences | Allele-selective ASO — lowers only the mutant copy, spares the healthy one | Phase 1b/2a done | SELECT-HD | On hold — seeking partner | ~46% mutant-huntingtin reduction (2024–25); pivotal trial not filed. Takeda exited 2024. Wave |
| VO659Vico Therapeutics | ASO targeting the CAG repeat itself (also tested in two ataxias) | Phase 1/2a | NCT05822908 | Ongoing — dosing redesigned | ~38% mutant-protein reduction (n=3); switched to twice-yearly dosing after nerve-inflammation cases. US sites planned late 2026. HDBuzz |
| TominersenRoche / Genentech | Non-selective ASO, spinal injection; lowers all huntingtin | Phase 2 — failed | GENERATION HD2 | Discontinued Jul 2026 | Lowered huntingtin & improved biomarkers but did not slow disease at 16 mo. Also failed Phase 3 in 2021. HDBuzz |
| RG6496Roche | Next-gen allele-selective ASO | Phase 1 — halted | POINT-HD | Discontinued Jul 2026 | Stopped after 3 participants over animal repeat-dosing findings (not human harm). HDBuzz |
Pills taken by mouth that change how the huntingtin gene's message is processed, so the cell destroys it. Reach the whole body; no injections.
| Program | Mechanism | Phase | Trial | Status | Latest readout |
|---|---|---|---|---|---|
| Votoplam (PTC518)Novartis / PTC | Oral splicing modulator; lowers huntingtin body-wide | Phase 3 | NCT07326709 INVEST-HD |
Recruiting ~770 people · US sites open |
24-mo open-label data (Apr 28, 2026) showed dose-dependent slowing vs. natural history. Phase 3 answer ~3 yrs out. PTC |
| SKY-0515Skyhawk | Oral splicing modifier; lowers huntingtin and PMS1 (DNA-repair gene) | Phase 2/3 | NCT06873334 FALCON-HD |
Active · expanding AU/NZ enrolled; US/CA/UK sites opening |
Final 15-mo data (Sep 15, 2026): ~60% mutant-huntingtin lowering, ~25% PMS1 reduction, cUHDRS +0.94 vs −0.65 external — but n=15, open-label, no placebo. FALCON-HD target raised to 600. HDBuzz |
| BranaplamNovartis | Oral splicing modulator (repurposed from an SMA drug) | Phase 2b — halted | VIBRANT-HD | Discontinued 2022 | Stopped after signs of nerve damage (peripheral neuropathy). Full results in Nature Medicine, 2025. |
A single brain surgery delivers a virus carrying instructions that make brain cells produce less huntingtin, permanently. Irreversible — and the source of 2025's landmark result.
| Program | Mechanism | Phase | Trial | Status | Latest readout |
|---|---|---|---|---|---|
| AMT-130uniQure | One-time AAV gene therapy, surgically infused; a microRNA lowers huntingtin | Phase 1/2 → BLA | NCT04120493 NCT05243017 |
Closed to enrollment FDA application submitted Sep 2, 2026 |
48-mo data (Sep 29, 2026): slowing held on daily function (TFC 61%, p=0.008) but the combined cUHDRS scale missed significance (44%, p=0.144); n=12 high-dose, open-label vs. external controls. uniQure. Earlier 36-mo cUHDRS data (80% slower decline vs. external controls, nominal p=0.005) is the basis of the BLA — submitted Sep 2, 2026, not yet accepted or approved. |
| SPK-10001 (RG6662)Roche / Spark | One-time AAV gene therapy, infused into caudate & putamen; lowers huntingtin | Phase 1/2 | NCT06826612 | Recruiting 5 US sites |
First patient dosed June 2025; safety-focused, very slow enrollment. No efficacy data. Continues despite Roche ending its ASO programs. HDBuzz |
| AB-1001AskBio / Bayer | AAV gene therapy delivering the CYP46A1 enzyme (brain-cholesterol pathway) — not huntingtin-lowering | Phase 1/2 | NCT05541627 | Active · Not Recruiting Single site, Paris |
No efficacy readout published. Small, open-label, single-site. |
| VY-HTT01Voyager | AAV gene therapy (huntingtin-lowering) | Preclinical — dropped | VYTAL (never enrolled) | Discontinued 2021 | Discontinued before dosing anyone; company pivoted to a delivery-technology platform. |
The newest frontier. Instead of lowering huntingtin, these aim to stop the CAG mutation from growing inside brain cells over a lifetime — the process now thought to drive when and how fast HD strikes. Every dedicated program here is preclinical: none has been given to a human yet.
| Program | Mechanism | Phase | Sponsor | Status | Latest milestone |
|---|---|---|---|---|---|
| LQT-23 | Oral MSH3 (DNA-repair) inhibitor; aims to slow CAG expansion | Preclinical | LoQus23 | IND filing targeted late 2026 | Named as candidate Jan 6, 2026. First human trial 2027 at the earliest. Release |
| RGT-0474060 | Oral PMS1-lowering splice modulator | Preclinical | Rgenta | IND-enabling; clinic targeted 2027 | Unveiled at CHDI conference Feb 25, 2026; data not peer-reviewed. Release |
| LTS-201 | One-time AAV gene therapy that knocks down MSH3 in brain | Preclinical | Latus Bio | IND targeted Q3 2026 | $97M raise May 4, 2026. "50 years of benefit" is a computer model, not observed data. Release |
| MSH3 gene therapy | AAV delivering a microRNA to suppress MSH3 | Preclinical | Passage Bio | Candidate selection expected 2H 2026 | Earliest-stage of the group — no candidate named yet. Q4 update |
| HRN-001 / ASO1025 | ASO that raises FAN1, a protein that protects against expansion | Preclinical | Harness Therapeutics | Trials targeted 2027 | FAN1 biology less settled than MSH3. Candidate name differs across sources. HDSA pipeline |
Note: SKY-0515 (in the oral-pill table) also lowers PMS1, making it the only compound touching this pathway that's actually in patients — but that PMS1 effect is secondary and unproven in humans. A Pfizer MSH3 inhibitor and ReviR's RTX317 appear on HDSA's pipeline list but couldn't be independently confirmed, so they're treated here as unverified.
These help with symptoms — chorea (involuntary movements), mood, cognition — but do not slow HD. The three approved drugs are today's standard of care; everything a person can get at a pharmacy right now is here.
| Program | For | Phase | Sponsor | Status | Notes |
|---|---|---|---|---|---|
| Valbenazine (Ingrezza) | Chorea | Approved | Neurocrine | FDA approved | Approved Aug 18, 2023. Once-daily, no dose titration. Symptomatic only. |
| Deutetrabenazine (Austedo) | Chorea | Approved | Teva | FDA approved | Approved Apr 3, 2017. Boxed warning for depression/suicidality in HD. |
| Tetrabenazine (Xenazine) | Chorea | Approved | Generic | FDA approved | Approved Aug 15, 2008. First HD chorea drug; boxed warning for depression/suicidality. |
| SOM3355 (bevantolol) | Chorea | Phase 2b done | SOM Biotech | Phase 3 planned | FDA/EMA aligned on a single Phase 3 (2025); start slipped to late 2026 / early 2027. Phase 2b not yet peer-reviewed; benefit seen in a subgroup. HDBuzz |
| PridopidinePrilenia / Ferrer | Function / progression | Phase 3 (retry) | Prilenia | Failed once; re-testing | PROOF-HD missed its endpoint (2023, published 2025); EU application withdrawn Nov 2025. PRECISE-HD retry began Jul 7, 2026 in a narrow subgroup. Nat Med |
| Dalzanemdor (SAGE-718) | Cognition | Phase 2 — failed | Sage | Discontinued 2024 | DIMENSION trial missed its endpoint (Nov 19, 2024); program ended. There is still no approved treatment for HD cognitive decline. HDBuzz |
A non-scientist's guide to the ideas behind the pipeline. Nothing here is dumbed down — it's the same science, in everyday words.
Everyone has a gene called huntingtin (HTT). In HD, one copy carries a stutter — a DNA sequence "CAG" repeated too many times. That faulty copy makes a misshapen huntingtin protein that slowly damages brain cells, especially in a region called the striatum that controls movement, mood and thinking. HD is inherited: a child of an affected parent has a 50% chance of inheriting the expanded gene. Most therapies in development attack this problem at one of three points — the faulty message, the harmful protein, or the process that makes the stutter grow.
The dominant idea for a decade: if the mutant gene makes a harmful protein, make less of it. Some drugs (ASOs, siRNA) are injected and mop up the gene's "message" (RNA) before it becomes protein. Others (oral splicing pills) change how that message is built so the cell discards it. A gene therapy does the same thing permanently with one surgery.
The catch: Roche's tominersen lowered huntingtin substantially and still didn't help patients. Lowering the protein is necessary, but on its own may not be enough — or may need to happen earlier, or only to the mutant copy.
Healthy huntingtin does important jobs in the brain, so lowering both copies may carry a cost. "Allele-selective" drugs are engineered to silence only the mutant copy and leave the healthy one working. Wave's WVE-003 is the furthest along, but its pivotal trial is on hold pending a partner.
A one-time treatment. Surgeons infuse a harmless virus deep into the brain; it carries genetic instructions that tell brain cells to keep producing a huntingtin-lowering molecule for years. The upside is "one and done"; the downside is it's irreversible brain surgery, and if the dose or approach is wrong there's no undo. AMT-130 is the leading example.
The newest and fastest-moving idea. That CAG "stutter" isn't fixed — in vulnerable brain cells it keeps growing across a person's life, and the leading model is that cells get sick only once it passes a high threshold. Certain DNA-repair genes (MSH3, PMS1, FAN1) influence how fast it grows. Drugs that tune those genes might slow — or delay — the disease at its root, even before symptoms start. All still preclinical.
Measurements that track the disease without waiting years for symptoms to change. NfL (neurofilament light) is a protein that leaks from damaged neurons — it rises before symptoms and is used to see whether a drug is affecting the brain. Crucially, NfL is a signal, not proof of benefit: tominersen improved NfL and still failed. The HD-ISS is a research staging framework that lets trials enroll people at the earliest biological stage, before diagnosis — it isn't something a clinic uses to predict an individual's course.
None of the above is approved to slow HD yet. What is available treats symptoms: three FDA-approved drugs (valbenazine, deutetrabenazine, tetrabenazine) reduce chorea, the involuntary movements of HD. They don't touch the underlying disease, and some carry warnings around mood — but they meaningfully improve daily life, and are managed by an HD specialist alongside physical, occupational, speech and mental-health care.
One more term worth knowing — cUHDRS. Most trials now report the "composite Unified HD Rating Scale," a single number blending movement, thinking, and daily function. When you read "75% slowing on cUHDRS," it means the treated group's combined score worsened much less than the comparison group's over the same period.
Type your city below to see the nearest HD specialty centers and interventional trial sites, ranked by distance — it works for any US location. If your town isn't listed, choose the nearest larger city. The first call to make is to an HD center, not to a trial sponsor; the center confirms eligibility, handles referrals, and knows which sites are actually open this month.
Distances are straight-line, measured city-center to city-center from the city you select — not from your street address — and real driving distance is longer. Site lists verified Oct 12, 2026 from ClinicalTrials.gov mirrors (INVEST-HD 51 sites, Novartis page Sep 22, 2026; PRECISE-HD registry Sep 17, 2026; FALCON-HD Sep 17, 2026). Age ranges shown per trial are from each study’s eligibility criteria; final eligibility is always decided by the trial site at screening; early-rollout lists change monthly, so confirm with the site. Enroll-HD (the observational registry) has many more locations — use the Enroll-HD site finder. For live trial matching by ZIP code with eligibility screening, use HDSA’s HD Trialfinder — it stays current automatically and includes a call center. HDSA Center of Excellence list is per hdsa.org (Kaiser Permanente CA, a statewide partner, is omitted from distance ranking).
Here's one city worked through in full, as an illustration of how to read what the finder gives you — type your own city above for yours. The short version for Baltimore: it's an unusually well-served corner of the map for HD. There's an HDSA Center of Excellence in the city itself, a second about an hour away in Washington DC, and the Northeast Corridor means "an hour away" is a train ride, not a road trip.
| Center | Where | What it is | Distance |
|---|---|---|---|
| Johns Hopkins HD Center | Baltimore, MD | HDSA Center of Excellence. Maryland's only HD Center of Excellence; multidisciplinary care within Psychiatry & Behavioral Sciences. | In the city |
| MedStar Georgetown HD Center | Washington, DC | HDSA Center of Excellence. Directed by Dr. Karen Anderson; also the DC-area INVEST-HD trial site. | ~38 mi (~1 hr) |
| Penn HD Center | Philadelphia, PA | HDSA Center of Excellence. Runs the region's gene-therapy trial site. | ~106 mi (~2 hr) |
Verified on hdsa.org, Oct 12, 2026. Johns Hopkins is the in-town option; Georgetown (DC) is the second-closest Center of Excellence. HDSA Centers of Excellence at UVA (Charlottesville, ~170 mi) and VCU (Richmond, ~150 mi) are farther alternatives.
| Trial | Ages | Type | Nearest site | Reachable from Baltimore? | Link |
|---|---|---|---|---|---|
| INVEST-HD (votoplam)Phase 3 · oral pill | 21–70 | Interventional | Johns Hopkins, Baltimore — recruiting (newly open, Sep 2026) | Yes — in Baltimore As of Sep 22, 2026 this oral trial is recruiting at Johns Hopkins itself (site contacts Jee Bang, Etta Bernard) — the nearest option is now in town, no longer the DC drive. An oral pill also means fewer intensive on-site procedures than surgery or spinal injections. Georgetown DC (~38 mi) remains a backup. | Recruiting page ↗ |
| Enroll-HDobservational registry | Any adult observational |
Observational | Johns Hopkins, Baltimore — recruiting | Yes — in Baltimore Not a drug trial: an annual check-in study that powers HD research worldwide (and provided the comparison data for AMT-130). A concrete local way to contribute and stay in line for future trials; ask the Hopkins HD Center. | Site finder ↗ |
| SPK-10001Phase 1/2 · gene therapy | 25–65 | Interventional | UPenn, Philadelphia (~106 mi) & U Pittsburgh (~247 mi) — recruiting | Reachable, but a big step Philadelphia is a ~2-hr drive or ~1-hr train. But this is one-time brain surgery with intensive follow-up — a far larger commitment than a pill, and its age cap is 65. | Recruiting page ↗ |
| PRECISE-HD (pridopidine)Phase 3 · oral pill | 23–65 | Interventional | Johns Hopkins is a listed site but not yet recruiting; nearest open site Wichita, KS | Local site not open The Johns Hopkins site hasn't opened for this trial yet (registry, Sep 17, 2026); 5 of 28 US sites now recruiting (nearest: Columbus OH). It also excludes people on certain movement drugs, and caps at age 65. | Recruiting page ↗ |
| FALCON-HD (SKY-0515)Phase 2/3 · oral pill | 25+ no max |
Interventional | No open US site yet (nearest listed: Englewood, CO — not yet open) | Not yet US expansion is underway but no site is open near Baltimore. Worth re-checking; no upper age limit when it does open. | Recruiting page ↗ |
| ALN-HTT02Phase 1 · siRNA injection | 25–70 | Interventional | Canada / UK / Germany — no US sites | Not accessible No US sites as of Oct 12, 2026. | Recruiting page ↗ |
Trial statuses verified Oct 12, 2026, via ClinicalTrials.gov and its mirrors; ages shown are each study's eligibility range, but final eligibility (disease stage, function, medications) is decided by the site at screening. Early-rollout site lists change often — confirm with the site before making plans. Useful companions: HDSA’s HD Trialfinder (live US trial matching by ZIP, with a call center), the Enroll-HD site finder, and the European Huntington Association’s HDTrialFinder.
From Baltimore, Johns Hopkins is in town and Georgetown/DC is ~38 miles — about an hour by car on I-95, or a frequent MARC Penn Line / Amtrak ride between Baltimore Penn Station and DC Union Station (~40 min–1 hr). Philadelphia (~2 hr by car, ~1 hr by Amtrak) is the backup; Pittsburgh is the long one at ~250 miles. That easy DC corridor is exactly why the oral INVEST-HD trial at Georgetown is the most livable fit for someone near Baltimore; an HD center can confirm what suits your situation.
The trusted places this tracker draws from — worth knowing directly. Each does something this page deliberately doesn't try to replace.
| Resource | What it's for |
|---|---|
| HDSA HD Trialfinder | Live US clinical-trial matching by ZIP code, with eligibility screening and a call center staffed by real people. The best first stop for "which trial could I actually join?" |
| HDSA Centers of Excellence | The directory of HD specialty clinics nationwide — comprehensive care teams, social workers, and the front door to most trials. HDSA helpline: 1-800-345-HDSA. |
| HDBuzz | Plain-language HD research news written by scientists — the gold standard for honest, hype-free reporting on every new result. |
| ClinicalTrials.gov | The official US trial registry — every trial's design, sites, contacts, and recruiting status, straight from the source. |
| Enroll-HD | The worldwide HD observational study (22,000+ participants) — an annual check-in that powers research everywhere; joining is one concrete way to contribute. Site finder. |
| CHDI Foundation | The nonprofit research foundation behind much of the HD drug-discovery science and the field's major conferences. |
| Huntington Study Group | The clinical-research network of HD credentialed investigators and coordinators that runs many US trials. |
| HDYO | The Huntington's Disease Youth Organization — age-appropriate support and education for young people in HD families. |
| EHA HDTrialFinder | The European Huntington Association's trial finder — the equivalent resource for family in Europe. |