Support Innovation in HD Drug Development, HD History 

Online Survey: Supporting Innovation in HD Drug Development  

Provide the Food and Drug Administration, researchers, medical product developers and other stakeholders with input about the challenges of living with HD by participating in the HD Symptoms and Treatment Impact Survey. This first-of-its-kind survey has been created by the HD community, for the HD community, to inform the FDA’s decisions and oversight during drug development. To learn more and take the survey, click here.  

HDSA Externally-led Patient Focused Drug Development Meeting 

The survey mentioned above will serve as the foundation for an HDSA Externally-led Patient Focused Drug Development Meeting for Pre-Symptomatic and Early to Mid-Stage Adult Onset HD. Join us in-person or virtually for an important opportunity to tell the FDA, researchers and medical product developers about your experiences living with pre-symptomatic or early to mid-stage HD as well as your preferred treatment outcomes for new therapies. This meeting will be hosted by HDSA on November 13 from 1:00-5:00 PM at the Samuel Riggs IV Alumni Center, University of Maryland, College Park, MD and virtually. It will include two panels comprised of people living with pre-symptomatic, early and mid-stage HD and family members who will share their stories. The rest of the meeting is devoted to providing you a forum to share your own experiences. Those attending virtually will have a chance to participate and share their perspectives as well. Click here to learn more about the meeting, or click here to apply for the opportunity to attend the meeting either in person or virtually.  

This Month in HD History 

HD is caused by an expansion of an area in the huntingtin gene called the polyglutamine tract, made up of CAG repeats in the DNA coding of the gene. When HD is passed down from one generation to the next, the length of the CAG repeat can sometimes increase a little (or a lot). This is important because the onset of symptoms is influenced by CAG repeat length. It’s variable and depends on many other factors, but more CAG repeats can lead to an earlier onset of HD. A person with an expansion in the polyglutamine tract of their HD gene will sometimes pass down more CAG repeats to their child. This phenomenon is called anticipation, and it’s part of the reason that people in the same family sometimes get symptoms at such different ages.  

In August of 1995, researchers studied hundreds of families and published key findings about differences in the HD gene between parents and children. They found that when dads with HD passed the gene to their children, the CAG number was more likely to jump much higher between generations, sometimes leading to Juvenile Huntington’s Disease (JHD). Understanding this aspect of HD inheritance has helped to better guide the diagnosis of JHD. If you want a deeper look, the original published article can be read here.