Huntington's Disease Foundation

06/18/2026

Nancy Wexler's vision continues to inspire the future of Huntington's disease research. The Nancy S. Wexler Discovery Fund supports the next generation of researchers working to advance treatments - and ultimately a cure - for HD.

Learn more or make a gift here: https://hdfoundation.org/nancy-s-wexler-discovery-fund/

06/17/2026

Letter to the HD Community from Daniel Leonard, uniQure (6/17/2026)

06/17/2026

In an exciting update from uniQure, we’ve learned that they have plans to submit a Biologics License Application (BLA) to the FDA by September of this year. Ahead of that submission, they will align with the agency on the design of a confirmatory study, including the best control group to confirm the ability of AMT-130 to modify the course of Huntington’s disease. If the BLA is approved, it would allow uniQure to market AMT-130 for the treatment of Huntington’s disease while the confirmatory study runs, under the understanding that if additional data proves the drug does not significantly modify disease progression, it will be pulled from the market. This is an exciting day for the HD community because it opens the door for AMT-130 to advance in the United States, but this does not mean that AMT-130 has been approved or is broadly accessible. We’re eagerly awaiting the outcome from the BLA submission in Q3 and looking forward to seeing what the FDA-aligned confirmatory study design will look like.

We’d like to acknowledge the momentum generated by the HD community in recent months as we move closer to having a disease modifying therapy in hand. The willingness of the FDA to work with rare disease communities, like ours, is a welcome partnership as we cross the finish line.

https://uniqure.gcs-web.com/node/12796/pdf

-130 HDYO (Huntington's Disease Youth Organisation) HDBuzz Factor-H American Brain Coalition Philly Cure HD (Huntington's Disease) Erin Paterson Huntington Study Group Huntington Society of Canada Champions for HD European Huntington Association Rare Patient Voice

06/16/2026

Nancy Wexler, HDF President and leader in the HD community, was recently profiled in The New York Times.

We invite you to read it here:

https://www.nytimes.com/2026/06/11/science/researcher-huntingtons-disease-wexler.html?unlocked_article_code=1.pVA.vvNZ.3epINJHhZXox&smid=url-share

Photo Credit: Jackie Molloy for The New York Times

HDYO (Huntington's Disease Youth Organisation) HDBuzz Factor-H American Brain Coalition Philly Cure HD (Huntington's Disease) Erin Paterson Huntington Study Group Huntington Society of Canada Champions for HD European Huntington Association Rare Patient Voice

Her decades of work on Huntington’s disease helped lead to the creation of a genetic test for the devastating condition. Why didn’t she take it herself?

06/15/2026

Congratulations to Sarah Tabrizi who was recognized in the King’s Birthday Honours for her outstanding contributions to Huntington’s disease!

Congratulations to members of UCL’s community who have been recognised in the King’s Birthday Honours for their outstanding contributions to education, Huntington’s disease, children's oral health and anti-doping in sport.

06/11/2026

The Huntington’s Disease Foundation is proud to announce Won-Seok Lee, PhD, Broad Institute of MIT and Harvard, as the 2026 recipient of the Nancy S. Wexler Young Investigator Prize.

This Prize honors the pioneering spirit, relentless dedication, and enduring optimism of Nancy S. Wexler, PhD, President of the Huntington's Disease Foundation. It is awarded annually to an early career researcher whose work reflects the highest caliber of excellence, diligence, and creative thinking.

Dr. Lee's research targets one of the most pressing open questions in HD: how do neurons with very long somatic CAG repeat expansions actually die? To answer this question, he’s developed a project that intersects the genetic changes that cause HD with how that impacts protein production.

Learn more about Dr. Lee's work: https://hdfoundation.org/nancy-s-wexler-prize -awardee

HDYO (Huntington's Disease Youth Organisation) HDBuzz Factor-H American Brain Coalition Philly Cure HD (Huntington's Disease) Erin Paterson Huntington Study Group Huntington Society of Canada Champions for HD European Huntington Association Rare Patient Voice Broad Institute of MIT and Harvard

06/05/2026

Dr. Sarah Hernandez, our Director of Research Programs, just wrapped an extraordinary 3-day immersion workshop in Boston for the next generation of HD scientists.

This year marked a milestone, as the first two days were dedicated to the inaugural cohort of HD-Career Advancement Grant recipients. These early-career researchers received intensive, hands-on training as they make the pivotal leap to independent principal investigators and the future leaders of HD research.

Day 3 brought our full young investigator community together for HDF's annual Young Investigator Scientific Workshop, uniting all these brilliant minds in one room to share their work, discuss the outstanding questions in the field, and drive HD research forward.

These enthusiastic, brilliant HD researchers inspire us. Supporting this next generation of scientific leaders is one of the many ways HDF is helping move research forward.

HDYO (Huntington's Disease Youth Organisation) HDBuzz Factor-H American Brain Coalition Philly Cure HD (Huntington's Disease) Erin Paterson Huntington Study Group Huntington Society of Canada Champions for HD European Huntington Association Rare Patient Voice Huntington's Disease Society of America

06/05/2026

Our recent Research Spotlight webinar, "A Legacy of Hope: The Essential Role of Brain Donation" is now available to watch anytime on our YouTube channel: https://youtu.be/5nt83ir-aBw?si=7YI4Xe7MOx30pbto

Dr. Dirk Keene and Dr. Dan Child joined us to share how they’re creating a new brain bank dedicated to Huntington's disease and what researchers can learn from it.

HDYO (Huntington's Disease Youth Organisation) HDBuzz Factor-H American Brain Coalition Philly Cure HD (Huntington's Disease) Erin Paterson Huntington Study Group Huntington Society of Canada Champions for HD European Huntington Association Rare Patient Voice University of Washington UC San Diego Health UC San Diego

Watch the Huntington's Disease Foundation Research Spotlight Webina...

06/04/2026

It's big news for the whole Huntington's disease community when an HD researchers is honored with such a prestigious award! Congratulations, Dr. Beverly Davidson!

"Throughout [Dr. Davidson's] career, she has focused on improving adeno-associated viruses (AAVs) for CNS gene therapies, with a particular emphasis now on HD...Her goal is to scale lab research in neurons, mouse models, and non-human primates (NHPs) to treat patients, including adults with HD."

https://www.genengnews.com/topics/genome-editing/asgct-2026-beverly-davidson-offers-vehicle-and-route-for-huntingtons-disease-gene-therapy/

Children's Hospital of Philadelphia American Society of Gene & Cell Therapy

Beverly Davidson, PhD, chief scientific strategy officer at Children's Hospital of Philadelphia, shares her research including a novel gene therapy approach for Huntington's disease.

06/03/2026

Exciting news for brain research!

The Allen Institute in Seattle says scientists have now learned enough about how the brain works to start fixing it when it breaks.