Top 5 Huntington’s disease news stories of 2025
HDSA convention news, new therapies among year's most-viewed topics
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The team at Huntington’s Disease News covered the latest community developments and updates in research related to Huntington’s disease throughout 2025.
We’ve compiled a list of the top five most-read articles we published in 2025. We look forward to continuing to serve the Huntington’s community in the years to come as a source for news and information.
No. 5 – HDSA 40th annual convention kicks off in Indianapolis
The Huntington’s Disease Society of America (HDSA) convention is an annual event that brings together people from throughout the Huntington’s community to share, learn, and join in hope for the future. Last year’s event, which was covered virtually by Huntington’s Disease News, took place June 26-28. The in-person conference was sold out, with many attendees tuning in virtually. The convention featured discussions on topics ranging from self-care and clinical trials to financial security and advocacy, and included the Team Hope Walk, support groups, and an award dinner and gala.
No. 4 – Experimental therapy AMT-130 scores FDA designation
uniQure announced in April that AMT-130, its experimental gene therapy for Huntington’s, received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA). This status is designed to expedite the development of treatments for serious diseases by providing regulatory benefits. AMT-130 aims to reduce levels of the mutant huntingtin (mHTT) protein that drives the disease. Data from two Phase 1/2 clinical trials, spanning up to three years, have shown that the therapy slows Huntington’s progression by 75%. While the FDA initially said that data from the trials could be used to support an application seeking AMT-130’s approval, the agency later said these were not sufficient.
No. 3 – Clinical testing moves forward for investigational therapy PTC518
PTC Therapeutics announced early last year that a Phase 2 clinical trial testing its investigational oral therapy PTC518 against a placebo in more than 250 adults with Huntington’s was nearing completion. The results, announced in May, showed that the treatment reduced huntingtin protein levels as designed, with signs of potential clinical benefit in participants with earlier-stage disease. PTC518’s development is now in the hands of Novartis.
No. 2 – HDSA 2025: Communication is key for navigating long-term care decisions
Experts at the HDSA convention discussed how long-term care facilities can benefit people with Huntington’s and offered guidance for families navigating emotionally charged discussions about care. The experts stressed the importance of clear communication — between patients and their caregivers, and between families and staff at care facilities — to ensure that everyone’s needs are being met. Working together can help make dealing with Huntington’s more manageable.
No. 1 – Huntington’s clinical trials discussed at HDSA convention
Several experimental treatments for Huntington’s disease are in clinical testing, and pharmaceutical representatives highlighted some of them during a session at the HDSA convention. Among them were uniQure’s AMT-130 and Roche‘s tominersen, which is currently being tested in a Phase 2 trial of early-stage Huntington’s patients. Also discussed were Roche’s experimental gene therapy, RG6662, which is in Phase 1/2 testing, and Wave Life Sciences’ WVE-003, which was shown to reduce mHTT levels and slow brain shrinkage in a Phase 1b/2a trial and is now slated for late-stage testing.
We hope these stories and all of our reporting in 2025 have been a useful resource for the Huntington’s community, and we look forward to continuing to be a resource in the years to come. We wish all our readers a bright and happy new year.
