Summary
- HDSA’s 39th Annual Convention
- uniQure granted RMAT designation for AMT-130
- HTT-lowering and Somatic Expansion: Can they be treated simultaneously?
- Community advocates talk about HD
HDSA’s 39th Annual Convention
Last week, HDSA welcomed over 750 members of the HD community to Spokane, WA for its 39th Annual Convention. The three days of learning and support around dozens of topics relevant to HD families included discussions on gene therapy, use of human tissue in HD research, HDSA-supported science, a Year in Review presentation from the HDBuzz team, and opportunities to Ask the Scientists Anything. Links to recorded sessions will be posted later this summer—stay tuned for more details in the near future!
uniQure granted RMAT designation for AMT-130
The FDA has given uniQure a special status called RMAT designation for the development of their HD gene therapy AMT-130. RMAT designation, which stands for “Regenerative Medicine Advanced Therapy,” provides uniQure the opportunity to meet more frequently with the FDA as their clinical trial continues. This designation does not indicate any positive or negative news related to the outcomes of the trial; it simply means that the FDA is interested enough in uniQure’s innovative approach to treating HD to warrant regular updates. If study data continues to support further development of AMT-130, this will help things move forward smoothly. More data from uniQure on the progress of the study is expected by the end of June. Read more from uniQure here.
HTT-lowering and Somatic Expansion: Can they be treated simultaneously?
Two strategies being explored to treat HD are lowering the amount of toxic huntingtin protein and slowing the expansion of CAG repeats in the huntingtin gene. Recent work from researchers at Massachusetts General Hospital indicates that some HTT-lowering drugs may also be able to slow the expansion of the huntingtin gene. The HD Buzz team broke down the findings of this study and what they mean in their most recent article. Read it in full here.
Community advocates talk about HD
The month of May might have ended, but HDSA is still celebrating the amazing work of community advocates to highlight HD. In honor of HD Awareness Month, HD community advocate and blogger, Gene Veritas, published an expose on a researcher who created a prominent model of HD. Learn more about Dr. X William Yang’s contributions to HD research here. Additionally, Albany chapter president and community advocate, Kathy Peake, talked HD with News10’s Ryan Peterson. Watch their conversation here.
