RareWays is a research directory. It helps you understand the science. It is not medical advice. Always discuss your care options with your healthcare team.
Coverage: 2020-01-01 onwards, with up to 300 results per query from PubMed and Europe PMC and separate limits on other sources. This is a selected index, not a complete literature search.
Updates are delayed or unverified. Check original sources for the latest information.
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Huntington's Disease: Research Summary
Printed from RareWays (rareways.org)
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Huntington's disease clinical trials update: August 2026.
Farag Mena et al., Journal of Huntington's disease (19 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42895253/
- 2.
Autonomic dysfunction in patients with Huntington's disease: Evidence from standardized cardiovascular reflex testing.
Carrozzo Giannicola et al., Journal of Huntington's disease (19 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42895481/
- 3.
Neuroprotective Role of Withaferin A in Huntington's Disease: Current Evidence and Future Perspectives.
Kumari Risha et al., Molecular neurobiology (17 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42891785/
- 4.
Disassembly of mitochondrial cristae architecture drives neurodegeneration in Huntington’s disease
Yutong Shang et al., Molecular Neurodegeneration (16 October 2026)
https://doi.org/10.1186/s13024-026-01005-4
- 5.
iPSC neurodegenerative disease initiative isogenic CAG repeat iPSC line for Huntington's disease.
Salazar Lisa et al., Journal of Huntington's disease (16 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42885744/
Clinical Trials : Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials. Each has its ID and full eligibility criteria at the link shown.
- 1.
A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease
NCT07536061 · Recruiting · Phase 1 · Sarepta Therapeutics, Inc.
Ages 21 to 70
https://clinicaltrials.gov/study/NCT07536061
- 2.
A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
NCT07326709 · Recruiting · Phase 3 · Novartis Pharmaceuticals
Ages 21 to 70
https://clinicaltrials.gov/study/NCT07326709
- 3.
A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers
NCT05509153 · Recruiting · Phase 2 · Western Sydney Local Health District
Ages 18 and over
https://clinicaltrials.gov/study/NCT05509153
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Huntington's Disease
Huntington's disease is a genetic condition that causes the gradual breakdown of nerve cells in the brain. It affects movement, thinking, and behaviour. Symptoms usually begin in mid-adulthood. There is currently no cure, but research into treatments is very active.
This overview is general information, not an individual medical assessment. Research is selected automatically and may include mismatches. How we select and explain research.
Most Recent Research
In this edition, we highlight several important developments across the Huntington's disease therapeutic pipeline. We discuss the launch and subsequent discontinuation of POINT-HD, the first-in-human phase I study of RG6496 (Hoffmann-La Roche), an allele-selective antisense oligonucleotide designed to selectively lower mutant huntingtin while largely preserving wild-type huntingtin expression. We also discuss the launch of two phase III efficacy trials: INVEST-HD, evaluating votoplam (Novartis Pharmaceuticals), and PRECISE-HD, evaluating pridopidine (Prilenia). In addition, we summarise recent advances across the field, including preclinical updates for LQT-23 (LoQus23), SRP-1005 (Sarepta Therapeutics) and HRN001 (Harness Therapeutics); interim 12-month clinical results from SKY-0515 (Skyhawk Therapeutics); the announcement by uniQure that 3-year data from the phase I/II AMT-130 study may support a Biologics License Application seeking accelerated approval and Roche's discontinuation of the phase II GENERATION HD2 study of tominersen. Finally, we provide an updated listing of registered and ongoing clinical trials in Huntington's disease.
Common Questions
What is Huntington's Disease?
Huntington's disease is a genetic condition that causes the gradual breakdown of nerve cells in the brain. It affects movement, thinking, and behaviour. Symptoms usually begin in mid-adulthood. There is currently no cure, but research into treatments is very active.
How many clinical trials are available for Huntington's Disease?
RareWays currently indexes 191 clinical trials for Huntington's Disease, of which 23 are actively recruiting. These counts cover indexed trials across countries, not just your country. An Australian site does not necessarily mean that site is recruiting. Check locations, eligibility and current availability with the study team.
Where does the research data for Huntington's Disease come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is refreshed automatically every day. All articles and trials link directly to their original sources.
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