HDSA Iowa Chapter

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

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

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