HDSA Orange County Chapter

Today’s announcement from uniQure marks an encouraging and meaningful step forward for the Huntington’s disease community.

The FDA’s indication that three-year data from the Phase I/II AMT-130 study may serve as the primary basis for a BLA for accelerated approval offers renewed hope to families who have been waiting far too long for treatment options.

This milestone reflects the power of a united HD community — families, advocates, clinicians, partner organizations, and Members of Congress — who helped ensure that the voices of those impacted by HD were heard.

While there is still important work ahead, including the regulatory process and a necessary confirmatory study, today brings hope and momentum.

HDSA remains committed to working with regulators, uniQure, HD sister organizations, clinicians, and the broader healthcare community to help move these next steps forward safely and as expeditiously as possible.

Read HDSA’s full statement: https://hdsa.org/news/hdsa-statement-on-uniqures-amt-130-regulatory-update/

Help for Today. Hope for Tomorrow.

HDSA Statement on uniQure’s AMT-130 Regulatory Update - Huntington's Disease Society of America HDSA Statement on uniQure’s AMT-130 Regulatory Update 1

In a significant reversal from its March 2026 position, the FDA has told uniQure that data already collected from AMT-130 clinical trials can support an expedited approval application in the United States.

The FDA also indicated openness to a follow-up trial where participants in the control group would continue on their current standard of care rather than undergoing a sham brain surgery, which would address a major ethical concern the HD community has raised.

uniQure plans to file for approval in the third quarter of 2026.

To read the full press release, click here:https://hdsa.org/wp-content/uploads/2026/06/PR_TypeB-Update_June-2026_06.17.26_Final-1.pdf and to read the Community Letter, visit:https://hdsa.org/wp-content/uploads/2026/06/2026-June-17-uniQure-Regulatory-Update_Community-Statement_Final.pdf

We had an amazing time at our 3rd annual Celebration of Hope Luncheon at the gorgeous Ocean Institute. Thank you to everyone who attended and enjoyed a day of community and inspiration.

On June 2, 2026, the Huntington’s Disease Society of America (HDSA) joined rare disease leaders, policy experts, patient advocates, and congressional offices for a congressional townhall briefing, “The Pathway to Cures and Treatments for Rare Diseases,” at the Rayburn House Office Building.

The event featured remarks and participation from Rep. Morgan Griffith and Rep. Jake Auchincloss, who joined advocates and rare disease leaders in discussing the importance of advancing meaningful pathways to treatments and cures for rare disease communities.

HDSA thanks the Congressional Rare Disease Caucus, Rep. Griffith, Rep. Auchincloss, congressional staff, patient advocates, and rare disease partners for their participation and commitment to advancing progress for rare disease communities.

To watch the full townhall briefing, visit: https://www.youtube.com/watch?v=EgBECsklK08

Our 2026 Symposium was a huge success! Our speakers were incredible, their presentations were exciting and informative, and everyone had a great time. Hope you were able to attend, and hope to see you at the next one!

For families impacted by Huntington's disease, every day brings challenges most people never see. A parent watching symptoms progress. A child living with uncertainty. A family holding onto hope for more answers and better treatments.

If you've been following along, sharing posts, or learning more about HD this month, now is the moment to turn awareness into impact. Visit, hdsa.org/hdawareness

Awareness creates understanding. Hope propels us forward. Real change happens when people choose to rise, speak up, lead, and make a difference.

Help support HDSA and help strengthen the programs, services, and community support that individuals and families affected by Huntington’s disease have come to rely on. When awareness meets action, hope becomes powerful.

Take Action. Go to: hdsa.org/hdawareness