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A welcoming home for HD research

Helping people make sense of Huntington's disease.

For families navigating a new diagnosis, caregivers looking for plain-language answers, researchers chasing the next hypothesis, and curious builders who want to see how AI can actually help. We pull the latest HD research every day, ground every answer in real papers, and link out to the people who know best.

Ask a question, in your language See how the Hub works

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.

How the Hub works

Pick a topic. Read what it means. Then do something with it.

Every HD topic on this page works the same way. Start with one that catches your eye, read the plain-language version, then jump straight into the papers, the hypotheses, or the open trials. Nothing here is a dead end.

1
Pick a topic

Choose one of the live HD topics below. These are the ideas the field is actually arguing about right now.

2
See what it means

Read a short, plain-language explanation: what it is, why it matters, what the evidence says, and what is still unclear.

3
Take an action

Show me papers, see AI hypotheses, find clinical trials, fetch more research. Every button is a real link, not a placeholder.

Therapeutic strategy

Huntingtin-lowering (ASOs and gene therapy)

A strategy worth watching Closest the field has come to disease-modifying

If the mutant huntingtin protein causes the damage, can we just make less of it? That is the simple question behind a whole family of HD programs, from antisense oligonucleotides delivered into the spinal fluid to one-time AAV gene therapies aimed at the striatum.

What it is

A set of therapies designed to reduce how much huntingtin protein the brain makes, using either ASOs, AAV gene therapy, or small molecules.

Why it matters

It targets HD upstream of symptoms. If it works, the same approach could slow the disease for many people rather than treating one symptom at a time.

What the evidence says

Early human data from AMT-130 has been encouraging enough for uniQure to plan regulatory filings, and several other programs are following close behind.

What is still unclear

How durable the effect is, which patients benefit most, and whether allele-selective approaches will end up safer than total HTT knockdown.

Do something with it
What this hub does

Four ways the Hub helps people

One website, one knowledge base, four different kinds of help. Whatever brought you here, there is a place to start.

forum
For families and curious learners

Understand the disease and navigate it

Ask plain-language questions and get answers grounded in real papers, with sources you can open and read. Available in English and 22 Indian languages. The moment a question crosses into personal medical territory, the system steps back and redirects to HDSA, HDBuzz, HDYO, and your own neurologist. We are not a clinic, on purpose.

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For data scientists and builders

Run the data and the papers yourself

Everything is open source and reproducible. The Kaggle notebook runs the same pipeline you see live, top-to-bottom on a fresh GPU in under ten minutes. Read real PubMed full text, parse figures, swap models, and add your own experiments. Apache 2.0 licensed.

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For researchers and reviewers

Generate new hypotheses, then stress-test them

An overnight loop walks 16 HD targets, nominates drug-repurposing candidates with explicit rationales, and scores them. Five experiments have shipped so far, each with an honest writeup of what worked and what did not. Spot the bad ideas. Champion the good ones.

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For everyone, working in the background

Scout new data automatically, every 24 hours

GitHub Actions runs the pipeline every morning. New PubMed papers, new ClinicalTrials.gov entries, fresh HDBuzz stories, refreshed Open Targets scores. The knowledge base gets a little sharper every day, without anyone having to remember to press refresh.

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What the Hub is tracking today
20
Recent Papers
30
Active Trials
20
Recruiting Now
16+
Known Targets

Updated May 16, 2026 at 09:26 AM. Auto-refreshed daily from PubMed, ClinicalTrials.gov, HDBuzz, and Open Targets.

What's Happening Now

HD News + Breakthroughs

More on HDBuzz open_in_new
Thu, 14 May 2026

Intruders in the Brain: What a Pig Model Reveals about Immune Cells in HD
Mon, 11 May 2026

The Great Care Conundrum: What “Good” Care Looks Like in Huntington’s Disease
Thu, 07 May 2026

A Second Path: uniQure Plans Regulatory Filing for AMT-130 in the UK
Mon, 04 May 2026

Two Years In: New Long-Term Extension Data from PIVOT-HD for Votoplam
Thu, 30 Apr 2026

April 2026: This Month in Huntington’s Disease Research
Fresh from PubMed

Latest Research Papers

All HD papers open_in_new
2026 Apr — Molecular therapy : the journal of the A
gene therapyprotein structure

Treatment of Huntington's disease with a pan-HTT-targeting CRISPR nuclease.

Huntington's disease (HD) is an inherited neurodegenerative disorder caused by an expansion of a CAG trinucleotide repeat in the huntingtin (HTT) gene, which leads to a mutant protein that destroys ne

PubMed open_in_new
2026 Jun — Disease models & mechanisms
protein structure

Induced pluripotent stem cells from a transgenic minipig model of Huntington's disease reveal early

Huntington's disease (HD) is a neurodegenerative autosomal dominant hereditary disease caused by a CAG triplet repeat expansion mutation in the gene encoding the huntingtin (HTT) protein. The main fea

PubMed open_in_new
2026 Apr — Cell chemical biology
autophagyprotein structure

Distinct autophagy impairment mechanisms of huntingtin aggregates with different polyQ lengths.

Huntington's disease (HD) is characterized by the aggregation of mutant huntingtin (mHTT) containing elongated polyglutamine (polyQ) tracts. mHTT aggregates that fail to be cleared by autophagy cause

PubMed open_in_new
2026 May — Laeknabladid
autophagyprotein structure

[Huntington´s disease - overview].

Huntington's disease (HD) is a rare, autosomal dominant neurodegenerative disorder characterized by progressive movement disturbances, psychiatric symptoms, and cognitive impairment. The disease is ca

PubMed open_in_new
2026 Apr — Journal of Huntington's disease
somatic expansion

Towards AI-driven prediction of HTT CAG size in super-expanded human spiny projection neurons from H

Somatic instability (SI) of the CAG tract in HTT is a major driver of neurodegeneration of Spiny Projection Neurons (SPNs), the primary neuronal subtype affected in Huntington's disease (HD). SPNs can

PubMed open_in_new
2026 Apr — Neurobiology of disease
gene therapyprotein structure

Propagation of gut-injected AAV2/1-Htt171-82Q to the brain induces Huntington's disease-like patholo

Huntington's disease (HD) is a progressive neurodegenerative disorder that leads to thbe production of pathogenic mutant huntingtin (mHtt) protein and is characterized by motor and psychiatric symptom

PubMed open_in_new
Reasons for Optimism

Active Clinical Trials

30 trials, 152,831 patients enrolled, 20 recruiting now

ClinicalTrials.gov open_in_new
NCT ID Title Sponsor Phase Status Intervention
NCT06774443 Hinting Task for Huntington's Disease University Medical Center N/A Recruiting Neuropsychological assessment
NCT07213648 Modification of Threat Interpretation Bias to Redu University of Virginia NA Recruiting MindTrails-Movement; Waitlist Control
NCT04012411 Study of BDNF Pathway Biomarkers in the Cerebrospi University Hospital, Mont NA Recruiting Brain MRI; Lumbar Punction; Blood sample
NCT07339514 A Phase I Clinical Study of ER2001 Injection for t ExoRNA Bioscience PHASE1, PHASE2 Active Not Recruiting ER2001 intravenous injection; Placebo
NCT02252380 ExAblate Transcranial MRgFUS for the Management of InSightec NA Active Not Recruiting Transcranial ExAblate System
NCT07246941 A Study to Evaluate the Safety, Tolerability, Phar Hoffmann-La Roche PHASE1 Recruiting RG6496; Placebo
NCT02855476 HDClarity: a Multi-site Cerebrospinal Fluid Collec University College, Londo N/A Recruiting
NCT06941662 Huntington's Disease Biobank: Advancing Remote Mon Stanford University N/A Recruiting computer vision, remote monitoring, bloo
NCT06546488 Cognitive Assessment Tools for Huntington's Diseas Ohio State University N/A Recruiting Assessments
NCT01574053 Enroll -HD: A Prospective Registry Study in a Glob CHDI Foundation, Inc. N/A Recruiting
Open Research

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_forward
Tocilizumab Exploring
Target: IL-6 / neuroinflammation

IL-6 receptor blockade may reduce inflammation from mutant huntingtin aggregation

Latest score: 80/100 Support: 0 Concerns: 0 Updated: 2026-03-29
Metformin Exploring
Target: mTOR / AMPK / autophagy

Promotes autophagy which may help clear mutant HTT aggregates

Latest score: 72/100 Support: 0 Concerns: 0 Updated: 2026-03-29
Rapamycin Exploring
Target: mTOR

mTOR inhibitor demonstrated clearance of mutant HTT in preclinical models

Latest score: 68/100 Support: 0 Concerns: 0 Updated: 2026-03-29
Riluzole Known/Tested
Target: Glutamate excitotoxicity

Glutamate release inhibitor, may reduce excitotoxicity in HD

Latest score: 60/100 Support: 0 Concerns: 1 Updated: 2026-03-29
Lithium Exploring
Target: TDP-43 / GSK-3β

May reduce TDP-43 phosphorylation and aggregation via GSK-3β inhibition, relevant to newly discovered HD-TDP-43 connection

Latest score: 55/100 Support: 0 Concerns: 0 Updated: 2026-03-29

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

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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.

Data Scientists + AI/ML Engineers

Run our autonomous research agents. Fork the repo. Try different models. Add experiments. This is open-source infrastructure you can build on.

Researchers + Domain Experts

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.

Students + Curious Builders

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.

For patients and families: This is not a medical resource. For support, guidance, and verified medical information, please go to HDSA, HDBuzz, or HDYO. They have doctors and scientists reviewing every word. We don't.

Trusted Organizations

Resources for Patients, Families + Researchers

These organizations are the real experts. We link to them because they do essential work.

HDSA
Support + Advocacy

Huntington's Disease Society of America. Find local support groups, social workers, Centers of Excellence. The primary advocacy organization in the US.
HDBuzz
Research News in Plain Language

Scientists explain HD research in words everyone can understand. The best place to follow new developments without needing a PhD.
HDYO
Young People

Huntington's Disease Youth Organization. Resources specifically for young people affected by HD — teens, young adults, and young families.
Enroll-HD
Join Research

The world's largest observational study for HD families. Your data helps researchers understand HD and design better clinical trials.
European HD Network
International Research

EHDN coordinates HD research across Europe. Clinical trials, registries, and working groups advancing the science globally.
HD Reach
Rehab + Exercise

Evidence-based rehabilitation exercises designed for people with HD. Physical therapy, speech therapy, and occupational therapy resources.
Make a Difference

Ways to Get Involved

Whether you're a patient, family member, researcher, developer, or just someone who cares.

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Join a Clinical Trial

20 HD trials are actively recruiting right now. Your participation directly accelerates the path to treatment.

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Contribute to Open Source

Our research agent, literature scanner, and drug repurposing tools are all open source. Data scientists, ML engineers, and bioinformaticians welcome.

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Share 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.

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Donate

Research funding saves lives. HDSA and CHDI Foundation fund the science that makes breakthroughs like AMT-130 possible.

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Share 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.

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