Helping people make sense of Huntington's disease.
An open HD research hub for families, learners, and researchers. See what is changing, understand why it matters, and ask questions against the actual papers and trials.
We are data scientists, not doctors. This is a research and education tool. For care decisions, please reach out to HDSA, HDBuzz, HDYO, or your own neurologist.
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Jump straight to the newest HD news, trial updates, and papers.
See what is recruiting now and which programs are moving.
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Curious about Huntington's? Start with the basics.
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menu_book Papers (PubMed)
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draft Preprints (biorxiv + medrxiv)
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newspaper HD news (HDBuzz)Mending the fabric of your genes via data_fetcher
26 experiments. Methods, findings and limitations.
Every tracked effort is an experiment: a hypothesis, a method, a result, and a published log. The cards below are the latest complete version of each one. Older runs stay in the audit trail and are marked superseded.
First-look LLM triage: 22 HD abstracts
The model analyzed 20 of 22 abstracts and surfaced recurring themes in huntingtin modifications, biomarkers and genetic mechanisms. These are exploratory literature signals, not clinical findings.
Full-text analysis of 16 HD research papers
The synthesis highlighted LIG1 repair fidelity, genetic interventions and mitochondrial-inflammatory connections. Proposed links remain model-generated hypotheses rather than established treatment effects.
Gemma 4 analysis of the 16-paper corpus
Gemma proposed connections among protein aggregation, mitochondrial dysfunction, inflammation and metabolic regulation. This second Experiment 3 publication offers another model's interpretation, not a validated ranking against Llama.
Expanded corpus: genetic and metabolic hypotheses
The report proposes a two-track framing of genetic damage alongside metabolic and immune dysfunction, highlighting copper homeostasis and gut-brain research as topics for further investigation.
June 2026 literature update
The synthesis highlighted membrane lipid interactions, UCHL3-related proteostasis and DNA-repair or epigenetic pathways. These signals motivated subsequent focused literature experiments.
Lipid disruption and protein clearance in HD
The model favored a lipid-first hypothesis connecting membrane organization, mitochondrial stress and protein clearance. The proposed order is a research question, not demonstrated causality.
TET1 and epigenetic regulation in HD
The model proposed TET1 suppression as a contributing factor that may interact with lipid and proteostasis pathways. Candidate compounds in the report are hypotheses for research, not evidence of benefit or safety in people.
Gut-brain and neuronal excitability hypotheses
The report proposes links among gut barrier function, systemic metabolism and neuronal pathways. It draws on Parkinson's disease analogies, which do not establish that HD begins in the gut.
NAD+ metabolism and mitochondrial research
The model highlighted NAD+ metabolism as a research direction and proposed precursor, PARP and sirtuin-related hypotheses. The report does not demonstrate that any candidate improves outcomes in HD.
A footwear concept for chorea-related instability
The October 7 concept revision starts at an illustrative 115 mm width, with the archived 174 mm width available separately. Broad straps, pull loops, open fastening and heel/forefoot cells remain untested design choices. Neither width is a fit recommendation. The archived 83% is a model ratio, not a measured benefit. No tested shoe or suitable personal dimensions have been established.
Qwen comparison: the saved run timed out
All 16 paper analyses and the cross-paper synthesis failed with an Ollama read timeout at localhost:11434. The file records input size only. No Qwen finding was produced.
Somatic CAG screen transcribed from the saved Gemma synthesis
The saved synthesis ranks MSH3, PMS1 and FAN1 and records 68 parsed analyses plus 7 JSON parse failures. Those ranks are the model's assessment, not a new measurement.
HDinHD access probe: no public catalog
Explorer redirects to a login page. The probe saved zero catalog rows. A normalizer and a synthetic fixture exist for when HDINHD_TOKEN is available.
Paper corpus catch-up after the May 5 stall
The scout corpus had stopped on 2026-05-05 at 86 papers while data_fetcher kept refreshing homepage papers. Discover-only search raised the corpus to 156 papers and analyzed none, because no LLM is configured.
Kynurenine Pathway Dysregulation in HD — KMO, IDO1, and QUIN/KYNA Imbalance
The script was not run. No papers were fetched and no model output was produced. This page is the finished account of that block.
Complement Cascade & Neuroinflammation in HD
The script was not run. No papers were fetched and no model output was produced. This page is the finished account of that block.
Cognitive Pharmacotherapy in Huntington's Disease
The script was not run. No papers were fetched and no model output was produced. This page is the finished account of that block.
SEMA4D Neuroinflammation Axis in Huntington's Disease
The script was not run. No papers were fetched and no model output was produced. This page is the finished account of that block.
Mitochondrial Membrane Stabilization in HD
The script was not run. No papers were fetched and no model output was produced. This page is the finished account of that block.
PDE10A Inhibitors — Striatal cAMP/cGMP Rescue in HD
The script was not run. No papers were fetched and no model output was produced. This page is the finished account of that block.
GLP-1R Agonists — Neuroprotection Repurposing for HD
The script was not run. No papers were fetched and no model output was produced. This page is the finished account of that block.
MPC Inhibition & ISR Normalization in HD
The script was not run. No papers were fetched and no model output was produced. This page is the finished account of that block.
Ferroptosis & Iron Chelation in HD
The script was not run. No papers were fetched and no model output was produced. This page is the finished account of that block.
cGAS-STING Innate Immune Pathway in HD
The script was not run. No papers were fetched and no model output was produced. This page is the finished account of that block.
Adenosine A2A Receptor Antagonism for HD MSN Protection
The script was not run. No papers were fetched and no model output was produced. This page is the finished account of that block.
Thiamine + Biotin TCA Cycle Rescue in HD
The script was not run. No papers were fetched and no model output was produced. This page is the finished account of that block.
What the shoe-building note actually connects to
The only HD shoe work found is Experiment #11, an unbuilt design study merged in PR 282. Named factories are capability examples, not partners. Other shoe hits in Ravi's repos are unrelated.
Superseded runs (kept for the audit trail, not featured)
Pick a topic. Read what it means. Then do something with it.
Huntingtin-lowering (ASOs and gene therapy)
Picture the huntingtin protein as a faulty thermostat that someone left turned up too high. Huntingtin-lowering programs try to turn the dial back down, so the brain has less of the harmful version to deal with in the first place.
A family of treatments that tries to make the brain produce less of the huntingtin protein. Some are repeat injections, some are a one-time gene therapy.
Snapshot recorded: October 15, 2026 at 01:07 PM (timezone not recorded). Refreshes scheduled daily; source results below.
PubMed: Refresh succeeded. Last successful refresh: October 15, 2026 at 01:07 PM (timezone not recorded). Last attempt: October 15, 2026 at 01:07 PM (timezone not recorded).
ClinicalTrials.gov: Refresh succeeded. Last successful refresh: October 15, 2026 at 01:07 PM (timezone not recorded). Last attempt: October 15, 2026 at 01:07 PM (timezone not recorded).
HDBuzz: Refresh succeeded. Last successful refresh: October 15, 2026 at 01:07 PM (timezone not recorded). Last attempt: October 15, 2026 at 01:07 PM (timezone not recorded).
Open Targets: Refresh succeeded. Last successful refresh: October 15, 2026 at 01:07 PM (timezone not recorded). Last attempt: October 15, 2026 at 01:07 PM (timezone not recorded).
HD News + Breakthroughs
Mending the fabric of your genes
Could rewiring the brain’s “brakes” improve movement in Huntington’s disease?
Your Voice Matters: Using Speech to Monitor Huntington’s Disease
Can’t Get You Out of My Head: The Huntington’s Disease Symptom That’s More Than Just Repetition
AMT-130 at Four Years: Hopeful Signs, Complex Controls
Latest Research Papers
Directing mutant huntingtin towards degradation: Strategies and therapeutic opportunities.
Huntington's Disease (HD) is a progressive neurodegenerative disease that is caused by a CAG-repeat expansion in the exon 1 of the huntingtin (HTT) gene, which results in the formation of toxic mutant
PubMed open_in_newNeurofilament Light Chain and Mutant Huntingtin Across Huntington's Disease Integrated Staging Syste
The Huntington's Disease Integrated Staging System (HD-ISS) provides a framework for staging HD but does not incorporate fluid biomarkers. The aim of this study was to examine neurofilament light chai
PubMed open_in_newUncovering hidden protein networks in Huntington's disease: implications for pathogenesis and therap
Huntington's Disease is a neurodegenerative disorder that progresses over time and can be passed down from parent to child. In uncontrolled motor activities, behavioural problems come into play, as we
PubMed open_in_newHuntingtin Aggregate-Responsive Autophagy Gene Circuit Mitigates Disease Pathology in R6/2 Mice.
The accumulation of mutant huntingtin (mHTT) aggregates drives the pathology of Huntington's disease (HD), yet therapies capable of distinguishing toxic species from wild-type proteins remain elusive.
PubMed open_in_newDefective membrane dynamics in Huntington's disease: A role of Rab11 dysfunction.
Huntington's disease (HD) is a life-threatening neurodegenerative disease caused by a mutation in Huntingtin (HTT). Neuropathology in HD is marked by a progressive loss of neurons in the caudate putam
PubMed open_in_newFluid and Imaging Biomarkers in Huntington's Disease.
Growing interest in earlier and mechanism-targeted interventions for Huntington's disease (HD) has brought focus on biomarkers capable of informing trial design and regulatory pathways. This review di
PubMed open_in_newActive Clinical Trials
30 tracked studies, 1,816 recorded as actual enrollment, 163,526 recorded as estimated enrollment, 19 recruiting now. These figures are the sum of the ClinicalTrials.gov enrollment fields in this table, not a count of unique patients.
| NCT ID | Title | Sponsor | Phase | Status | Enrollment | Enrollment type | Intervention |
|---|---|---|---|---|---|---|---|
| NCT07339514 | A Phase I Clinical Study of ER2001 Injection for t | ExoRNA Bioscience | PHASE1, PHASE2 | Active Not Recruiting | 27 | Estimated | ER2001 intravenous injection; Placebo |
| NCT02252380 | ExAblate Transcranial MRgFUS for the Management of | InSightec | NA | Active Not Recruiting | 10 | Estimated | Transcranial ExAblate System |
| NCT06585449 | A Study to Evaluate ALN-HTT02 in Adult Patients Wi | Alnylam Pharmaceuticals | PHASE1 | Recruiting | 66 | Estimated | ALN-HTT02; Placebo |
| NCT05822908 | A Safety and Pharmacokinetics Trial of VO659 in SC | Vico Therapeutics B. V. | PHASE1, PHASE2 | Recruiting | 68 | Estimated | VO659 |
| NCT06546488 | Cognitive Assessment Tools for Huntington's Diseas | Ohio State University | N/A | Recruiting | 76 | Estimated | Assessments |
| NCT06475898 | Longitudinal Endpoint Assessment of Disease Burden | Huntington Study Group | N/A | Recruiting | 600 | Estimated | |
| NCT06873334 | Study of SKY-0515 for Safety, Efficacy, and Pharma | Skyhawk Therapeutics, Inc | PHASE2, PHASE3 | Active Not Recruiting | 120 | Estimated | SKY-0515; SKY-0515 Placebo |
| NCT06626412 | Longitudinal SV2A PET and MRI in Premanifest HD | Universitaire Ziekenhuize | NA | Active Not Recruiting | 40 | Estimated | Diagnostic Test: 18F-SynVesT-1 PET; Volu |
| NCT00051324 | Neurobiological Predictors of Huntington's Disease | Jordan Schultz | N/A | Active Not Recruiting | 1,700 | Estimated | |
| NCT05509153 | A Randomised Controlled Trial, Of N-Acetyl Cystein | Western Sydney Local Heal | PHASE2 | Recruiting | 160 | Estimated | NAC; Placebo |
| NCT04713982 | Impact of Deutetrabenazine on Functional Speech an | Vanderbilt University Med | PHASE2, PHASE3 | Active Not Recruiting | 30 | Estimated | Deutetrabenazine |
| NCT06203106 | NYSCF Scientific Discovery Biobank | New York Stem Cell Founda | N/A | Recruiting | 10,000 | Estimated | Biological Sample Collection |
| NCT05360082 | Comparison Between [11C]UCB-J and [18F]SynVest-1 P | Universitaire Ziekenhuize | NA | Recruiting | 35 | Estimated | [11C]UCB-J PET; [18F]SynVest-1 PET |
| NCT01574053 | Enroll -HD: A Prospective Registry Study in a Glob | CHDI Foundation, Inc. | N/A | Recruiting | 35,000 | Estimated | |
| NCT05243017 | Safety and Efficacy of AMT-130 in European Adults | UniQure Biopharma B.V. | PHASE1, PHASE2 | Active Not Recruiting | 14 | Actual | intra-striatal rAAV5-miHTT |
| NCT02994719 | Gait Analysis in Neurological Disease | Beth Israel Deaconess Med | N/A | Active Not Recruiting | 120 | Estimated | Anti-Parkinson medication; Deep Brain St |
| NCT06448546 | Gut Microbiomes in HD | University of Central Flo | N/A | Recruiting | 36 | Estimated | |
| NCT06147414 | Development of Non-Invasive Prenatal Diagnosis for | Assistance Publique - Hôp | N/A | Recruiting | 550 | Estimated | Blood sample |
| NCT06626308 | Early Biomarkers in Premanifest Huntington's Disea | University Hospital, Gren | NA | Recruiting | 20 | Estimated | brain functional MRI |
| NCT06941662 | Huntington's Disease Biobank: Advancing Remote Mon | Stanford University | N/A | Recruiting | 200 | Estimated | computer vision, remote monitoring, bloo |
| NCT06876857 | University of Central Florida CereBank | University of Central Flo | N/A | Recruiting | 50,000 | Estimated | |
| NCT07537075 | An Extension of SKY-0515 in Participants With Hunt | Skyhawk Therapeutics, Inc | PHASE2, PHASE3 | Enrolling By Invitation | 500 | Estimated | SKY-0515 |
| NCT04012411 | Study of BDNF Pathway Biomarkers in the Cerebrospi | University Hospital, Mont | NA | Recruiting | 135 | Estimated | Brain MRI; Lumbar Punction; Blood sample |
| NCT02855476 | HDClarity: a Multi-site Cerebrospinal Fluid Collec | University College, Londo | N/A | Recruiting | 2,500 | Estimated | |
| NCT03434548 | IMarkHD: in Vivo Longitudinal Imaging of HD Pathol | King's College London | N/A | Recruiting | 113 | Estimated | PET imaging; Multi-modal MRI imaging |
| NCT06209515 | Sociodemographic Factors and Criminal Behaviour Pr | Kuopio University Hospita | N/A | Active Not Recruiting | 61,292 | Estimated | |
| NCT05596760 | Promoting Goals-of-Care Discussions for Patients W | University of Washington | NA | Active Not Recruiting | 1,802 | Actual | Jumpstart Guide |
| NCT05808153 | Innovative Imaging and Cognitive BIOmarkers to Pre | Assistance Publique - Hôp | NA | Recruiting | 80 | Estimated | radiotracer injection |
| NCT06634628 | iMagemHTT-009- FIH Evaluation of Novel Mutant Hunt | CHDI Foundation, Inc. | EARLY_PHASE1 | Recruiting | 27 | Estimated | Radioligand [11C]CHDI-00491009 |
| NCT07451613 | Safety and Tolerability Study of Human Neural Stem | Leslie Thompson | PHASE1, PHASE2 | Recruiting | 21 | Estimated | hNSC-01 |
Published Research Ideas
AI-assisted repurposing ideas with explicit uncertainty. These are starting points for review, not recommendations or validated findings.
Read Experiment #1: Full Analysis arrow_forwardNew June 2026 knock-in pig study (PMID 42322611): mHTT disrupts TBP binding to the TET1 promoter, silencing TET1 transcription before symptom onset. TET1 converts 5mC to 5hmC (DNA demethylation); its loss causes aberrant
Oligomer inhibitor shown to ameliorate HD pathology in mouse and cellular models. Exp 6 signal: mHTT hijacks neuronal lipid rafts; anle138b stabilizes oligomer conformation before raft association. Preclinical confidence
Exp 7 identified CYP46A1 activation as the highest-confidence early intervention: restoring cholesterol catabolism may stabilize lipid raft microdomains disrupted by mHTT, preventing the downstream proteostasis cascade.
New June 2026 paper: selectively targeting HTT1a isoform provides superior molecular rescue vs. lowering canonical HTT. HTT1a is enriched in disease-vulnerable neurons. Isoform-selective lowering preserves full-length HT
HDBuzz Jun 25 2026: PROTACs act as molecular glue, recruiting E3 ubiquitin ligases to tag mutant huntingtin (mHTT) for proteasomal destruction while leaving wild-type HTT intact. Unlike ASOs/siRNA (which lower all HTT mR
New June 2026 paper: somatic CRISPR knockout of Msh3 mitigates HD pathology in mice. Msh3 drives somatic CAG repeat expansion in neurons — without it, the repeat stays stable. This is a one-time intervention that targets
Important: These hypotheses are triage artifacts, not evidence of efficacy. They have not been clinically validated, experimentally confirmed, or expert-reviewed unless explicitly stated.
These cards are generated from data/hypotheses_tracker.json, not hand-picked homepage copy. Are you an HD researcher? We'd love your feedback. Review and discuss on GitHub
Who This Is For
This is best viewed as open research infrastructure: a place where data scientists, AI engineers, bioinformaticians, and researchers can test whether agent workflows actually help with literature review, hypothesis triage, and research communication.
Run our autonomous research agents. Fork the repo. Try different models. Add experiments. This is open-source infrastructure you can build on.
Review our AI-generated hypotheses. Spot what's promising and what's wrong. Your expertise is what turns computational ideas into real science. Join the discussion.
Want to learn how AI applies to drug discovery? Read our experiment reports — we show every step, what worked, and what didn't. No PhD required to follow along.
Resources for Patients, Families + Researchers
These organizations are the real experts. We link to them because they do essential work.
Huntington's Disease Society of America. Find local support groups, social workers, Centers of Excellence. The primary advocacy organization in the US.
Scientists explain HD research in words everyone can understand. The best place to follow new developments without needing a PhD.
Huntington's Disease Youth Organization. Resources specifically for young people affected by HD — teens, young adults, and young families.
The world's largest observational study for HD families. Your data helps researchers understand HD and design better clinical trials.
EHDN coordinates HD research across Europe. Clinical trials, registries, and working groups advancing the science globally.
Evidence-based rehabilitation exercises designed for people with HD. Physical therapy, speech therapy, and occupational therapy resources.
Ways to Get Involved
Whether you're a patient, family member, researcher, developer, or just someone who cares.
Join a Clinical Trial
19 HD trials are actively recruiting right now. Your participation directly accelerates the path to treatment.
Find recruiting trials open_in_newContribute to Open Source
Our research agent, literature scanner, and drug repurposing tools are all open source. Data scientists, ML engineers, and bioinformaticians welcome.
View on GitHub open_in_newShare Your Story
Patient and family stories drive awareness and funding. Share your experience with HDSA or on social media to help others feel less alone.
HDSA Get Involved open_in_newDonate
Research funding saves lives. HDSA and CHDI Foundation fund the science that makes breakthroughs like AMT-130 possible.
Donate to HDSA open_in_newShare Feedback or Ideas
Are you an HD researcher? See something interesting in our hypotheses? Spot an error? We want to hear from you — every correction makes this better.
Join the Discussion open_in_new